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HomeMy WebLinkAbout04.17.2024 Board Correspondence - FW_ Evidence Review of Adverse Effects (4)From:Clerk of the Board To:York, Danette Cc:Thompson, William; Lee, Lewis Subject:Board Correspondence - FW: Evidence Review of Adverse Effects (2) Date:Wednesday, April 17, 2024 11:44:52 AM Attachments:NAM2.pdf Please see Board Correspondence - From: lance dreiss <lancedreiss@att.net> Sent: Wednesday, April 17, 2024 9:53 AM To: acasler@sustainabilitymc.com; Connelly, Bill <BConnelly@buttecounty.net>; assemblymember.gallagher@assembly.ca.gov; Teeter, Doug <DTeeter@buttecounty.net>; ca01dl.outreach@mail.house.gov; Waugh, Melanie <mwaugh@buttecounty.net>; Ring, Brian <bring@buttecounty.net>; pcbs@countyofplumas.com; Stephens, Brad J. <BStephens@buttecounty.net>; Durfee, Peter <PDurfee@buttecounty.net>; sheriff@pcso.net; Kimmelshue, Tod <TKimmelshue@buttecounty.net>; preyinghawkreport@gmail.com; Clerk of the Board <clerkoftheboard@buttecounty.net>; Ritter, Tami <TRitter@buttecounty.net>; davidhollister@countyofplumas.com; District Attorney <District_Attorney@buttecounty.net>; Pickett, Andy <APickett@buttecounty.net>; senator.dahle@senate.ca.gov Subject: Evidence Review of Adverse Effects (2) .ATTENTION: This message originated from outside Butte County. Please exercise judgment before opening attachments, clicking on links, or replying.. Public Record https://urldefense.com/v3/__https://ipak-edu.org/NAM2? utm_source=substack&utm_medium=email__;!!KNMwiTCp4spf!A2Sk3TrzVAssRjdcti5XGIl4UeZbCYM 0wvVJA4G4tiiUqggzFAZ4IGjTCm_jVXmAWP13K_TMDmgfrlwXSSvjXIrbsL-kxw$ diana dreiss CONTRIBUTORS DETAILS All downloadable National Academies titles are free to be used for personal and/or non-commercial academic use. Users may also freely post links to our titles on this website; non-commercial academic users are encouraged to link to the version on this website rather than distribute a downloaded PDF to ensure that all users are accessing the latest authoritative version of the work. All other uses require written permission. (Request Permission) This PDF is protected by copyright and owned by the National Academy of Sciences; unless otherwise indicated, the National Academy of Sciences retains copyright to all materials in this PDF with all rights reserved. Visit the National Academies Press at nap.edu and login or register to get: – Access to free PDF downloads of thousands of publications – 10% off the price of print publications – Email or social media notifications of new titles related to your interests – Special offers and discounts SUGGESTED CITATION BUY THIS BOOK FIND RELATED TITLES This PDF is available at http://nap.nationalacademies.org/27746 Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration (2024) 336 pages | 8.5 x 11 | PAPERBACK ISBN 978-0-309-71832-5 | DOI 10.17226/27746 Anne R. Bass, Kathleen Stratton, Ogan K. Kumova, and Dara Rosenberg, Editors; Committee to Review Relevant Literature Regarding Adverse Events Associated with Vaccines; Board on Population Health and Public Health Practice; Health and Medicine Division; National Academies of Sciences, Engineering, and Medicine National Academies of Sciences, Engineering, and Medicine. 2024. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration. Washington, DC: The National Academies Press. https://doi.org/10.17226/27746. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. Consensus Study Report Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Anne R. Bass, Kathleen Stratton, Ogan K. Kumova, and Dara Rosenberg, Editors Committee to Review Relevant Literature Regarding Adverse Events Associated with Vaccines Board on Population Health and Public Health Practice Health and Medicine Division PREPUBLICATION COPY—Uncorrected Proofs Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREPUBLICATION COPY: Uncorrected Proofs NATIONAL ACADEMIES PRESS 500 Fifth Street, NW Washington, DC 20001 This activity was supported by a contract between the National Academy of Sciences and Health Resources and Services Administration, which includes funds from the Centers for Disease Control and Prevention. Any opinions, findings, conclusions, or recommendations expressed in this publication do not necessarily reflect the views of any organization or agency that provided support for the project. International Standard Book Number-13: 978-0-309-XXXXX-X International Standard Book Number-10: 0-309-XXXXX-X Digital Object Identifier: https://doi.org/10.17226/27746 This publication is available from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu. Copyright 2024 by the National Academy of Sciences. National Academies of Sciences, Engineering, and Medicine and National Academies Press and the graphical logos for each are all trademarks of the National Academy of Sciences. All rights reserved. Printed in the United States of America. Suggested citation: National Academies of Sciences, Engineering, and Medicine. 2024. Evidence review of the adverse effects of COVID-19 vaccination and intramuscular vaccine administration. Washington, DC: The National Academies Press. https://doi.org/10.17226/27746. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREPUBLICATION COPY: Uncorrected Proofs The National Academy of Sciences was established in 1863 by an Act of Congress, signed by President Lincoln, as a private, nongovernmental institution to advise the nation on issues related to science and technology. Members are elected by their peers for outstanding contributions to research. Dr. Marcia McNutt is president. The National Academy of Engineering was established in 1964 under the charter of the National Academy of Sciences to bring the practices of engineering to advising the nation. Members are elected by their peers for extraordinary contributions to engineering. Dr. John L. Anderson is president. The National Academy of Medicine (formerly the Institute of Medicine) was established in 1970 under the charter of the National Academy of Sciences to advise the nation on medical and health issues. Members are elected by their peers for distinguished contributions to medicine and health. Dr. Victor J. Dzau is president. The three Academies work together as the National Academies of Sciences, Engineering, and Medicine to provide independent, objective analysis and advice to the nation and conduct other activities to solve complex problems and inform public policy decisions. The National Academies also encourage education and research, recognize outstanding contributions to knowledge, and increase public understanding in matters of science, engineering, and medicine. Learn more about the National Academies of Sciences, Engineering, and Medicine at www.nationalacademies.org. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREPUBLICATION COPY: Uncorrected Proofs Consensus Study Reports published by the National Academies of Sciences, Engineering, and Medicine document the evidence-based consensus on the study’s statement of task by an authoring committee of experts. Reports typically include findings, conclusions, and recommendations based on information gathered by the committee and the committee’s deliberations. Each report has been subjected to a rigorous and independent peer-review process and it represents the position of the National Academies on the statement of task. Proceedings published by the National Academies of Sciences, Engineering, and Medicine chronicle the presentations and discussions at a workshop, symposium, or other event convened by the National Academies. The statements and opinions contained in proceedings are those of the participants and are not endorsed by other participants, the planning committee, or the National Academies. Rapid Expert Consultations published by the National Academies of Sciences, Engineering, and Medicine are authored by subject-matter experts on narrowly focused topics that can be supported by a body of evidence. The discussions contained in rapid expert consultations are considered those of the authors and do not contain policy recommendations. Rapid expert consultations are reviewed by the institution before release. For information about other products and activities of the National Academies, please visit www.nationalacademies.org/about/whatwedo. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. COMMITTEE v PREPUBLICATION COPY: Uncorrected Proofs COMMITTEE TO REVIEW RELEVANT LITERATURE REGARDING ADVERSE EVENTS ASSOCIATED WITH VACCINES GEORGE J. ISHAM (Chair), Senior Fellow, HealthPartners Institute ANNE R. BASS (Vice Chair), Professor of Clinical Medicine, Department of Medicine, Division of Rheumatology, Hospital for Special Surgery, Weill Cornell Medicine ALICIA CHRISTY, Professor of Obstetrics and Gynecology, Uniformed Services University; Adjunct Professor, Howard University School of Medicine DELISA FAIRWEATHER, Professor of Medicine, Director of Translational Research, Department of Cardiovascular Medicine; Codirector of Research for the Ehlers-Danlos Syndrome Clinic, Department of General Internal Medicine, Mayo Clinic (Jacksonville, FL) JAMES S. FLOYD, Codirector, Cardiovascular Health Research Unit, Associate Professor of Medicine, Adjunct Professor of Epidemiology, University of Washington ERIC J. HEGEDUS, Professor and Chair, Department of Rehabilitation Sciences Tufts University School of Medicine CHANDY C. JOHN, Ryan White Professor of Pediatrics, Professor of Medicine, Microbiology and Immunology, Director, Ryan White Center for Pediatric Infectious Diseases and Global Health, Indiana University School of Medicine JOHN EDWARD KUHN, Schermerhorn Professor of Orthopaedic Surgery, Chief of Shoulder Surgery, Department of Orthopaedic Surgery, Vanderbilt University Medical Center EVAN MAYO-WILSON, Associate Professor; Department of Epidemiology, University of North Carolina Gillings School of Global Public Health THOMAS LEE ORTEL, Chief, Division of Hematology, Department of Medicine Professor of Medicine and Pathology, Duke University School of Medicine NICHOLAS S. REED, Assistant Professor, Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health; Assistant Professor, Department of Otolaryngology, Division of Otology/Audiology, Johns Hopkins University School of Medicine ANDY STERGACHIS, Professor and Associate Dean of Pharmacy, School of Pharmacy; Professor of Global Health, School of Public Health, University of Washington MICHEL TOLEDANO, Assistant Professor of Neurology, Department of Neurology, Mayo Clinic (Rochester, MN) ROBERT B. WALLACE, Irene Ensmenger Stecher Professor Emeritus of Epidemiology and Internal Medicine, University of Iowa OUSSENY ZERBO, Research Scientist II, Vaccine Study Center, Division of Research Kaiser Permanente Northern California National Academy of Medicine Fellow INMACULADA HERNANDEZ, Professor, San Diego Skaggs School of Pharmacy and Pharmaceutical Sciences, University of California Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. vi VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs Health and Medicine Division Staff KATHLEEN STRATTON, Study Director OGAN K. KUMOVA, Program Officer (since February 2023) DARA ROSENBERG, Associate Program Officer NERISSA HART, Senior Program Assistant (through May 2023) OLIVIA LOIBNER, Senior Program Assistant (since June 2023) MISRAK DABI, Finance Business Partner REBECCA MORGAN, Senior Research Librarian ANNE-MARIE HOUPPERT, Senior Research Librarian ROSE MARIE MARTINEZ, Director, Board on Population Health and Public Health Practice Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. REVIEWERS vii PREPUBLICATION COPY: Uncorrected Proofs Reviewers This Consensus Study Report was reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise. The purpose of this independent review is to provide candid and critical comments that will assist the National Academies of Sciences, Engineering, and Medicine in making each published report as sound as possible and to ensure that it meets the institutional standards for quality, objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. We thank the following individuals for their review of this report: DOUGLAS B. CINES, Director, Coagulation Laboratory; Director, Office of Faculty Development, Pathology and Laboratory Medicine; Professor of Pathology and Laboratory Medicine (Hematology-Oncology), University of Pennsylvania School of Medicine BETTY DIAMOND, Director, Director, Institute of Molecular Medicine, The Feinstein Institute for Medical Research North Shore-LIJHealth System, Northwell Health KATHRYN EDWARDS, Professor of Pediatrics; Sarah H. Sell and Cornelius Vanderbilt Chair, Vanderbilt Vaccine Research Program; Vanderbilt University Medical Center MARIE GRIFFIN, Professor Emerita, Vanderbilt University School of Medicine AKIKO IWASAKI, Howard Hughes Medical Institute Investigator; Director, Center for Infection and Immunity; Sterling Professor of Immunobiology and Molecular, Cellular, and Developmental Biology, Yale University EMILY JUNGHEIM, Chief of Reproductive Endocrinology and Infertility in the Department of Obstetrics and Gynecology, Northwestern University TIANJING LI, Associate Professor, University of Colorado Anschutz Medical Campus JENNIFER S. LIN, Distinguished Investigator, Kaiser Permanente Center for Health Research CLAUDIA LUCCHINETTI, Dean, Dell Medical School; Senior Vice President for Medical Affairs, University of Texas at Austin H. CODY MEISSNER, Professor of Pediatrics and Medicine, Geisel School of Medicine at Dartmouth; Senior Vaccine and Biologics Development Analyst, Biomedical Advanced Research and Development Authority; Administration for Strategic Preparedness and Response, U.S. Department of Health and Human Services Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. viii VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs BRIAN OLSHANSKY, Emeritus Professor of Internal Medicine - Cardiovascular Medicine, Carver College of Medicine, University of Iowa, University of Iowa Hospital and Clinics JAMES SEGARS, Director, Division of Reproductive Sciences and Women’s Health Research, Johns Hopkins University School of Medicine UMASUTHAN SRIKUMARAN, Assistant Professor, Orthopaedic Surgery, Johns Hopkins University GRETA C. STAMPER, Audiology Division Chair, Audiology Externship Program Director, Consultant in Otorhinolaryngology, Mayo Clinic Although the reviewers listed above provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations of this report, nor did they see the final draft before its release. The review of this report was overseen by coordinator DAVID SAVITZ, Professor of Epidemiology, Brown University, and monitor WALTER FRONTERA, Professor of Physical Medicine, Rehabilitation, and Sports Medicine, University of Puerto Rico School of Medicine. They were responsible for making certain that an independent examination of this report was carried out in accordance with the standards of the National Academies and that all review comments were carefully considered. Responsibility for the final content rests entirely with the authoring committee and the National Academies. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. ACKNOWLEDGEMENTS ix PREPUBLICATION COPY: Uncorrected Proofs Acknowledgments The Committee to Review Relevant Literature Regarding Adverse Events Associated with Vaccines and the committee staff would like to thank many individuals for their contributions throughout all phases of the study: Misrak Dabi (Finance Business Partner), Crysti Park (Program Coordinator), Lori Brenig (Editorial Projects Coordinator), Taryn Young (Report Review Associate), Leslie Sim (Senior Report Review Officer), Benjamin Hubbert (Communications Specialist), Amber McLaughlin (Director of Communications), Tasha Bigelow (copy editor), Rebecca Morgan (Senior Research Librarian), and Anne Marie Houppert (Senior Research Librarian). The committee acknowledges and thanks the members of the public who provided valuable insight to the committee via email correspondence and in public comments. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. x VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. CONTENTS xi PREPUBLICATION COPY: Uncorrected Proofs Contents Preface xvii Acronyms and Abbreviations xix Summary 1 1 Introduction 13 2 Immunologic Response to COVID-19 Vaccines 27 3 Neurologic Conditions and COVID-19 Vaccines 53 GUILLAIN-BARRÉ SYNDROME ....................................................................................... 53 CHRONIC INFLAMMATORY DEMYELINATING POLYNEUROPATHY .................... 72 BELL’S PALSY ..................................................................................................................... 76 TRANSVERSE MYELITIS ................................................................................................... 86 CHRONIC HEADACHE........................................................................................................ 92 POSTURAL ORTHOSTATIC TACHYCARDIA SYNDROME.......................................... 95 4 Sensorineural Hearing Loss, Tinnitus, and COVID-19 Vaccines 113 SENSORINEURAL HEARING LOSS ................................................................................ 113 TINNITUS ............................................................................................................................ 122 5 Thrombosis with Thrombocytopenia Syndrome, Immune Thrombocytopenic Purpura, Capillary Leak Syndrome, and COVID-19 Vaccines 133 THROMBOSIS WITH THROMBOCYTOPENIA SYNDROME ...................................... 133 IMMUNE THROMBOCYTOPENIC PURPURA ............................................................... 141 CAPILLARY LEAK SYNDROME ..................................................................................... 149 6 Vascular Conditions and COVID-19 Vaccines: Myocardial Infarction, Stroke, Pulmonary Embolism, Deep Vein Thrombosis and Venous Thromboembolism 157 MYOCARDIAL INFARCTION .......................................................................................... 164 ISCHEMIC STROKE ........................................................................................................... 170 HEMORRHAGIC STROKE ................................................................................................ 175 Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. xii VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs DEEP VEIN THROMBOSIS, PULMONARY EMBOLISM, AND VENOUS THROMBOEMBOLISM ..................................................................................................... 181 7 Myocarditis, Pericarditis, and COVID-19 Vaccines 199 8 Sudden Death and COVID-19 Vaccines 239 9 Female Infertility and COVID-19 Vaccines 245 10 Shoulder Injuries and Vaccines 257 SUBACROMIAL/SUBDELTOID BURSITIS .................................................................... 259 ACUTE ROTATOR CUFF OR ACUTE BICEPS TENDINOPATHY ............................... 264 CHRONIC ROTATOR CUFF DISEASE ............................................................................ 268 ADHESIVE CAPSULITIS ................................................................................................... 269 SEPTIC ARTHRITIS ........................................................................................................... 273 BONE INJURY .................................................................................................................... 275 AXILLARY OR RADIAL NERVE INJURY ...................................................................... 279 PARSONAGE-TURNER SYNDROME .............................................................................. 282 COMPLEX REGIONAL PAIN SYNDROME (CRPS) ....................................................... 288 11 Crosscutting Remarks 299 Appendix A Committee Member and Staff Biographies 311 Appendix B 317 Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. BOXES, FIGURES, AND TABLES xiii PREPUBLICATION COPY: Uncorrected Proofs Boxes, Figures, and Tables BOXES S-1 Statement of Task, 2 S-2 Categories of Causation, 4 1-1 Statement of Task, 15 3-1 Conclusions for Guillain-Barré Syndrome, 53 3-2 Conclusions for Chronic Inflammatory Demyelinating Polyneuropathy, 72 3-3 Conclusions for Bell’s Palsy, 76 3-4 Conclusions for Transverse Myelitis, 86 3-5 Conclusions for Chronic Headache, 92 3-6 Conclusions for Postural Orthostatic Tachycardia Syndrome, 95 4-1 Conclusions for Sensorineural Hearing Loss, 113 4-2 Conclusions for Tinnitus, 122 5-1 Conclusions for Thrombosis with Thrombocytopenia Syndrome, 133 5-2 Conclusions for Immune Thrombocytopenic Purpura, 141 5-3 Conclusions for Capillary Leak Syndrome, 149 6-1 Conclusions for Myocardial Infarction, 164 6-2 Conclusions for Ischemic Stroke, 170 6-3 Conclusions for Hemorrhagic Stroke, 175 6-4 Conclusions for Deep Vein Thrombosis, Pulmonary Embolism, and Venous Thromboembolism, 181 7-1 Conclusions for Myocarditis and Pericarditis, 199 8-1 Conclusions for Sudden Death, 239 9-1 Conclusions for Female Infertility, 245 10-1 Conclusions for Shoulder Injuries, 258 11-1 Conclusions Regarding BNT162b2, 300 11-2 Conclusions Regarding mRNA-1273, 301 11-3 Conclusions Regarding Ad26.COV2.S, 301 Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. xiv VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs 11-4 Conclusions Regarding NVX-CoV2373, 302 11-5 Conclusions Regarding Shoulder Injuries, 303 11-6 Conclusions for Which the Evidence Establishes a Causal Relationship, 304 11-7 Conclusions for Which the Evidence Favors Acceptance of a Causal Relationship, 304 11-8 Conclusions for Which the Evidence Favors Rejection of a Causal Relationship, 305 FIGURES 2-1 COVID-19 vaccines contributing to this report and their mechanism of action, 31 2-2 Immune responses to intramuscular administration of SARS-CoV-2 mRNA vaccines, 32 3-1 Overview of the pathogenesis and therapeutic targets of the two major Guillain-Barré syndrome subtypes, 55 3-2 Postulated mechanisms of orthostatic intolerance and tachycardia in POTS, 98 10-1 Illustration of intramuscular injection techniques, 260 TABLES S-1 Causal Conclusions Regarding COVID-19 Vaccines, 7 S-2 Conclusions Regarding Shoulder Injuries After to Any Vaccinations, 9 1-1 COVID-19 Vaccines Used in the United States, 16 1-2 COVID-19 Vaccine U.S. Food and Drug Administration Emergency Use Authorization Dates, Adults and Children, 19 2-1 Immune Responses to U.S. COVID-19 Vaccines, 37 2-2 Vaccine-Mediated Reactions and Their Mechanisms, 41 2-3 Most Commonly Used Adjuvants in Vaccines, 44 3-1 Epidemiological Studies in the Guillain-Barré Syndrome Evidence Review, 57 3-2 Pharmacovigilance Studies in the Guillain-Barré Syndrome Evidence Review, 66 3-3 Epidemiological Study in the Chronic Inflammatory Demyelinating Polyneuropathy, 74 3-4 Epidemiological Studies in the Bell’s Palsy Evidence Review, 78 3-5 Epidemiological Studies in the Transverse Myelitis Evidence Review, 88 3-6 Epidemiological Study in the Postural Orthostatic Tachycardia Syndrome Evidence Review, 98 4-1 Epidemiological Studies in the Sensorineural Hearing Loss Evidence Review, 117 4-2 Epidemiological Studies in the Tinnitus Evidence Review, 125 5-1 Epidemiological Studies in the Thrombosis with Thrombocytopenia Evidence Review, 137 Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. BOXES, FIGURES, AND TABLES xv PREPUBLICATION COPY: Uncorrected Proofs 5-2 Epidemiological Studies in the Immune Thrombocytopenic Purpura Evidence Review, 143 6-1 Epidemiological Studies in the Vascular Conditions Evidence Review, 162 6-2 Epidemiological Studies in the BNT162b2–Myocardial Infarction Evidence Review, 166 6-3 Epidemiological Studies in the mRNA-1273–Myocardial Infarction Evidence Review, 167 6-4 Epidemiological Study in the Ad26.COV2.S–Myocardial Infarction Evidence Review, 168 6-5 Epidemiological Studies in the BNT162b2–Ischemic Stroke Evidence Review, 172 6-6 Epidemiological Study in the mRNA-1273–Ischemic Stroke Evidence Review, 173 6-7 Epidemiological Study in the Ad26.COV2.S–Ischemic Stroke Evidence Review, 174 6-8 Epidemiological Studies in the BNT162b2–Hemorrhagic Stroke Evidence Review, 177 6-9 Epidemiological Study in the mRNA-1273–Hemorrhagic Stroke Evidence Review, 178 6-10 Epidemiological Study in the Ad26.COV2.S–Hemorrhagic Stroke Evidence Review, 179 6-11 Epidemiological Studies in the BNT162b2–Deep Vein Thrombosis Evidence Review, 183 6-12 Epidemiological Studies in the BNT162b2–Pulmonary Embolism Evidence Review, 185 6-13 Epidemiological Studies in the mRNA–1273–Pulmonary Embolism Evidence Review, 186 6-14 Epidemiological Study in the Ad26.COV2.S–Pulmonary Embolism Evidence Review, 187 6-15 Epidemiological Studies in the BNT162b2–Venous Thromboembolism Evidence Review, 188 7-1 Findings from Canada’s Strategy for Patient-Oriented Research, 209 7-2 Selected Epidemiological Studies of Risk of Myocarditis Associated with BNT162b2, 211 7-3 Selected Epidemiological Studies of Risk of Pericarditis Associated with BNT162b2, 214 7-4 Reports to VAERS After mRNA-Based COVID-19 Vaccination That Met the CDC’s Case Definition for Myocarditis Within a 7-Day Risk Interval per Million Doses of Vaccine Administered, 216 7-5 Selected Epidemiological Studies of Risk of Myocarditis Associated with mRNA-1273, 219 7-6 Selected Epidemiological Studies of Risk of Pericarditis Associated with mRNA-1273, 222 8-1 Epidemiological Study in the Sudden Death Evidence Review, 242 9-1 Clinical and Epidemiological Studies in the Female Infertility Evidence Review, 248 Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. xvi VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs 10-1 Case Reports Regarding Subacromial/Subdeltoid Bursitis After Vaccination, 261 10-2 Case Reports of Acute Rotator Cuff or Acute Biceps Tendinopathy After Vaccination, 265 10-3 Case Reports of Adhesive Capsulitis After Vaccination, 270 10-4 Case Reports of Septic Arthritis After Vaccination, 273 10-5 Case Reports of Bone Injury After Vaccination, 276 10-6 Case Reports of Axillary or Radial Nerve Injury After Vaccination, 280 10-7 Case Reports of Parsonage-Turner Syndrome After Vaccination, 284 10-8 Budapest Criteria to Diagnose Complex Regional Pain Syndrome, 288 10-9 Case Reports of Complex Regional Pain Syndrome After Vaccination, 289 Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREFACE xvii PREPUBLICATION COPY: Uncorrected Proofs Preface In the 4 years since the first case of COVID-19 was recognized and after a pandemic was declared by the World Health Organization three months later in March 2020, an estimated 3.5 million died from SARS-CoV-2 infection. Millions more became ill, and some have suffered long-term effects (“long COVID”) that are not yet understood fully. Aside from its health impact, the pandemic has caused marked social, economic, and political upheaval. We doubt any have had lives unchanged by COVID-19. The response to the pandemic has been extraordinary. By spring, 2021, only 1 year after the pandemic declaration, vaccines authorized by the U.S. Food and Drug Administration for emergency were being administered across the United States, indeed, around the world. It is estimated that more than 14 million lives were saved in the year after vaccines became available, with one death avoided for every 124 full vaccination courses. Lives were also saved by other public health interventions and often-heroic efforts of health care workers and health care systems. In the 3 years since vaccines against SARS-CoV-2 came into use, the safety and efficacy have been established. Booster vaccinations, and vaccines targeting new SARS-CoV-2 strains have been introduced and are now administered routinely alongside other vaccinations such as for influenza. While local, non-serious side effects, such as malaise or sore arm are seen as with any vaccine, in rare instances, serious adverse events thought to be linked to SARS-CoV-2 vaccination have been noted. The National Academies of Sciences, Engineering, and Medicine have long tackled challenging questions about vaccine safety, beginning with an assessment of the oral polio vaccine in 1977. When Congress enacted the National Childhood Vaccine Injury Act in 1986, it charged the Institute of Medicine (IOM) with reviewing the literature regarding adverse events associated with vaccines covered by the program. The IOM has addressed questions about the safety of routinely administered vaccines 11 times since then. Following in this tradition, the National Academies of Sciences, Engineering, and Medicine (the National Academies) tasked this consensus committee to assess the scientific evidence dispassionately regarding a list of harms potentially associated with vaccination against SARS-CoV-2, as well as an important potential harm associated with the administration of any vaccine, shoulder injury. Thanks to the extraordinary efforts of investigators around the world who rapidly pivoted their research efforts to focus on this new virus (including its treatment and prevention), we now have a large body of evidence to consider. However, despite that large body of evidence, our consensus committee found that in many, if not most, cases, the evidence was insufficient to accept or reject causality for a particular potential harm from a specific COVID-19 vaccine. In other cases, however, the committee considered the evidence to be sufficient to “favor rejection” or to “favor acceptance” of or establish causality. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. xviii VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs Limitations inherent in applying population-level average effects to draw conclusions about causes of specific events in individual subjects exist. For this reason, there is asymmetry in the committee’s conclusions, with options to conclude that the evidence “establishes a causal relationship,” “favors acceptance of causal relationship” or “favors rejection of a causal relationship,” but not one to “establish rejection of a causal relationship.” For every potential harm assessed, the committee evaluated the totality of evidence and did not apply what could be seen as arbitrary rules or thresholds regarding the number or types of studies required to draw conclusions. For the evaluation of select postulated vaccine harms, some study types were simply not available or were uninformative. For some cases, there was strong mechanistic as well as epidemiologic evidence supporting a causal relationship (e.g., thrombosis and thrombocytopenia syndrome), while, in others, the evidence was drawn largely from case reports. COVID-19 has, understandably, dominated headlines over the last three years, yet, routine vaccinations, such as, for seasonal influenza, are still given. The list of harms our committee was tasked to review were those for which HRSA had claims for compensation. Perhaps surprisingly, only a minority of these claims related to SARS-CoV-2 vaccination. In fact, over 60% of claims focused on shoulder injury associated with intramuscular vaccine administration. The term “SIRVA” (shoulder injury related to vaccine administration) has been introduced into the literature in recent years and was included in the committee’s statement of task. However, the term “SIRVA” encompasses many disparate shoulder conditions and, due to its lack of precision, the committee decided to dispense with this terminology. Instead, the committee addressed potential causal relationships between vaccine administration and specific shoulder related medical diagnoses (e.g., subacromial bursitis, radial nerve injury). This report does not address benefits of vaccination against SARS-CoV-2 or other pathogens, and readers will hopefully view causality findings in that broader context. Even when evidence of causality was established for some harms, the frequency of these harms was low. However, this report explicitly does not attempt to define point estimates for levels of risk. Many talented, knowledgeable individuals volunteered hours of their time to analyze and report the evidence. Initially strangers, the members of this committee worked through difficult methodological questions together, at times, engaging in spirited debate. In the process, we learned from one another, became a team, and friends. Equally important, members of that team were the committee staff, Dara Rosenberg, Ogan Kumova and Olivia Loibner, led by the incredibly wise and knowledgeable Kathleen Stratton and Rose Marie Martinez. The staff worked tirelessly every step of the way, providing indispensable support and guidance, and contributing greatly to the report itself. This is not the first IOM/National Academies report regarding vaccine safety. Nor will it be the last. We anticipate new vaccines and expect ongoing and future scientific research may challenge the findings reported here. This report necessarily reflects a snapshot in time, albeit a momentous one, and represents our best effort to report the truth. George J. Isham, Chair Anne R. Bass, Vice Chair Committee to Review Relevant Literature Regarding Adverse Events Associated with Vaccines Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. ACRONYMS AND ABBREVIATIONS xix PREPUBLICATION COPY: Uncorrected Proofs Acronyms and Abbreviations AAOS American Academy of Orthopedic Surgeons ACE2 angiotensin-converting enzyme 2 ADE acute demyelinating events AIDP acute inflammatory demyelinating polyneuropathy AMAN acute motor axonal neuropathy AMH anti-Müllerian hormone AV adenovirus vector BARDA Biomedical Advanced Research and Development Authority BP Bell’s Palsy bpm beats per minute CAR coxsackie and adenoviral receptor CDC Centers for Disease Control and Prevention CI confidence interval CICP Countermeasure Injury Compensation Program CIDP chronic inflammatory demyelinating polyneuropathy cMRI cardiac magnetic resonance imaging COVID-19 coronavirus disease 2019 CRP C-reactive protein CRPS complex regional pain syndrome CSF cerebrospinal fluid CSI corticosteroid injection DAMP damage-associated molecular pattern DCM dilated cardiomyopathy DNA deoxyribonucleic acid DTaP diphtheria, tetanus, and acellular pertussis vaccine DVT deep vein thrombosis ECG electrocardiogram EHR electronic health record EMG electromyogram EMR electronic medical record ESC European Society of Cardiology EUA emergency use authorization EV extracellular vesicle Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. xx VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs FDA Food and Drug Administration FSH follicle-stimulating hormone GBD global burden of disease GBS Guillain-Barré Syndrome HIT heparin-induced thrombocytopenia HLA human leukocyte antigen HPV human papillomavirus HR hazard ratio HRSA Health Resources and Services Administration HSV herpes simplex virus ICD International Classification of Diseases IFN interferon Ig immunoglobin IL interleukin IR incidence rate IRR incidence rate ratio ITP immune thrombocytopenic purpura IV intravenous IVF in vitro fertilization LH luteinizing hormone LNP lipid nanoparticle MI myocardial infarction MRI magnetic resonance imaging mRNA messenger ribonucleic acid MS multiple sclerosis NCS nerve conduction study NF155 neurofascin-155 NIH National Institutes of Health NR not reported NSAID nonsteroidal anti-inflammatory drug O:E observed to expected ratio OPV oral polio vaccine OR odds ratio PE pulmonary embolism POTS postural orthostatic tachycardia syndrome PPV positive predictive value PT physical therapy PTS Parsonage-Turner Syndrome Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. ACRONYMS AND ABBREVIATIONS xxi PREPUBLICATION COPY: Uncorrected Proofs RCT randomized controlled trial RI relative incidence RNA ribonucleic acid RR relative risk or risk ratio SARS-CoV-2 severe acute respiratory syndrome coronavirus-2 SC self-controlled SCCS self-controlled case series SD source data SHBG sex hormone binding globulin SIR standardized incidence ratio SIRVA shoulder injury related to vaccine administration SPOR Strategy for Patient-Oriented Research SSNHL sudden sensorineural hearing loss SSP supraspinatus SUD sudden unexpected death TM transverse myelitis TNF tumor necrosis factor TTH tension-type headache TTS thrombosis with thrombocytopenia syndrome VAERS Vaccine Adverse Event Reporting System VAS visual analogue scale VICP Vaccine Injury Compensation Program VITT vaccine-induced thrombotic thrombocytopenia VSD Vaccine Safety Datalink VTE venous thromboembolism WHO World Health Organization YLD years lived with disability YLL years of life lost Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. xxii PREPUBLICATION COPY: Uncorrected Proofs Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREPUBLICATION COPY—Uncorrected Proofs Summary Vaccines are a major public health success story, preventing or mitigating the effects of a myriad of infectious diseases. However, the threat of litigation over safety concerns related to the whole cell pertussis vaccines in particular led manufacturers to slow vaccine research and development and leave the market. In 1986, Congress addressed this looming crisis for public health by passing the National Childhood Vaccine Injury Act (P.L. 99-660) to improve federal coordination of vaccine efforts around research and development and address the concerns of those who asserted that they or their children were injured by vaccines. The Vaccine Injury Compensation Program (VICP), housed in the Health Resources and Services Administration (HRSA) in the Department of Health and Human Services and jointly administered by the Department of Justice, serves as a key policy solution developed by Congress. The program includes vaccines recommended for routine use in children or pregnant women, and anyone who receives those vaccines is eligible to apply for compensation. The VICP has long depended on the reports from the National Academies of Sciences, Engineering, and Medicine (the National Academies) as an important scientific contribution to its compensation decisions. HRSA also administers the Countermeasures Injury Compensation Program (CICP) for those harmed by medical countermeasures, which include vaccines, medications, devices, or other preventions, diagnostics, or treatments for a public health emergency or security threat. Established by the Public Readiness and Emergency Preparedness Act of 2005 (P.L. 148, Division C), CICP differs significantly from VICP (HRSA, 2023a). On January 31, 2020, the Secretary of Health and Human Services declared a Public Health Emergency related to SARS-CoV-2 under Section 319 of the Public Health Service Act. The public health emergency expired on May 11, 2023. The public health emergency was declared because SARS-CoV-2 and the disease caused by SARS-CoV-2, COVID-19, were the greatest public health crisis to date of the 21st century. As of February 2024, it had led to an estimated 7 million deaths worldwide, including 1.2 million deaths in the United States (WHO, 2024). COVID-19 was a major cause of death and illness in both adults and children. In 2021, COVID-19 was the third most common cause of death in adults in the United States (CDC, 2021), and from 2020–2022, COVID-19 was among the top 10 causes of death in children in the United States (Flaxman et al., 2023). Part of the public health emergency was the announcement of “Operation Warp Speed,” a rapid response by the federal government to speed vaccine development (for detailed information, see GAO, 2021). Four vaccines were developed and used in the United States, all under Emergency Use Authorization (EUA) (see FDA, 2023), with some now fully approved by the Food and Drug Administration (FDA). However, as of June 1, 2023, FDA revoked the EUA from Ad26.CoV2.S for safety concerns (FDA, 2023). EUA allowed vaccines to be used before all phase 3 trials were completed.1 COVID-19 vaccines, introduced in 2020, are highly effective in adults and children (CDC, 2023) and were key to control of the pandemic. COVID-19 1 The sentence was updated after the report was shared with the sponsor to clarify the EUA process. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 2 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs vaccines are estimated to have prevented 14.4 million deaths worldwide in the first year of vaccination alone (Watson et al., 2022). Although in this report the committee is tasked with evaluating the causal association with select serious harm, a comparative study analyzing the prevalence and types of side effects following COVID-19 vaccination showed that the most common side effects across different vaccines were flu-like syndrome and local reactions at the injection site, which aligns with the side effect profiles of many vaccines. STATEMENT OF TASK HRSA requested that the National Academies convene a committee to review the evidence regarding specific potential harms (see Box S-1 for the Statement of Task) related to the COVID-19 vaccines used in the United States. See Table S-1 for a list of the vaccines and naming conventions used in this report. The list of harms includes those for which, when the project began, HRSA had claims for compensation. The committee added postural orthostatic tachycardia syndrome (POTS) to its review after presentations at its second public meeting. HRSA also requested that the committee review the evidence regarding any vaccine, not specifically COVID-19 vaccines, and shoulder injuries, to help VICP better understand whether vaccination can cause very specific types of shoulder injuries or a more general syndrome that it designated as Shoulder Injuries Related to Vaccine Administration (Grimes, 2023). Claims regarding shoulder injuries after routinely administered vaccines are handled by VICP; COVID- 19 vaccines are currently the purview of CICP (HRSA, 2023b). For the committee’s work, it was irrelevant whether a vaccine is covered under VICP or CICP; the committee did not consider VICP or CICP processes when reviewing the evidence. BOX S-1 Statement of Task The National Academies of Sciences, Engineering, and Medicine will convene an ad hoc committee to review the epidemiological, clinical, and biological evidence regarding the relationship between • COVID-19 vaccines and specific adverse events i.e., Guillain-Barré Syndrome (GBS), chronic inflammatory demyelinating polyneuropathy (CIDP), transverse myelitis, Bell’s palsy, hearing loss, tinnitus, chronic headaches, infertility, sudden death, myocarditis/pericarditis, thrombosis with thrombocytopenia syndrome (TTS), immune thrombocytopenic purpura (ITP), thromboembolic events (e.g., cerebrovascular accident (CVA), myocardial infarction (MI), pulmonary embolism, deep vein thrombosis (DVT)), capillary leak syndrome, and • intramuscular administration of vaccines and shoulder injuries. The committee will make conclusions about the causal association between vaccines and specific adverse events. The National Academies convened an ad hoc committee comprising 15 members with expertise in epidemiology, causal inference, cardiology, rheumatology, gynecology, audiology, neurology, infectious disease, pediatrics, internal medicine, hematology, orthopedics, and Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. SUMMARY 3 PREPUBLICATION COPY—Uncorrected Proofs immunology. The committee held two sessions open to the public. On January 30, 2023, it heard from representatives of HRSA and CDC on how they intend to use its report and why they asked for the review. On March 30, 2023, the committee held an open session during which members of the public registered to provide 2-minute statements concerning its task. Although the committee reviewed the literature thoroughly, it did not conduct what is commonly referred to as a “systematic review,” the formal steps of which were described by IOM in 2011 (IOM, 2011). The processes and time frame for a systematic review were considered incompatible with this work and, more importantly, the goals were different from those of most systematic reviews and clinical guidelines. The committee was not tasked with estimating the magnitude or strength of associations between vaccinations and outcomes. To fulfill its narrower goals, the committee did incorporate important attributes of good systematic reviews, such as searching multiple databases, using structured search terms, prespecifying a final date of searching, and using multiple reviewers to screen out irrelevant abstracts identified in the search. The committee does not address the benefits of vaccines, which have been established for COVID-19 vaccines and all vaccines covered by VICP. This review addresses evidence only about specific potential harms and vaccines available in the United States. The committee does not make conclusions regarding specific patient cases (such as reported in published case reports) or whether VICP or CICP should award compensation in individual cases or in general. Vaccines and other medical products can cause both benefits and harms. Harms are sometimes described, including by previous IOM committees, using terms such as “adverse event,” “adverse effect,” “side effect,” or “safety.” Such terms might not convey the importance of unwanted medical events. Moreover, readers might be confused by the use of different terms with overlapping meanings or the same terms to mean different things in different contexts (Qureshi et al., 2022). For example, “adverse events” are defined in regulatory research as unwanted events not necessarily related to an intervention (e.g., a vaccine, a drug). By comparison, “adverse effects” are both unwanted and related to an intervention. On the other hand, “side effects” might be desirable or unwanted, and they are related to an intervention. Following best practices (Junqueira et al., 2023; Zorzela et al., 2016), this report uses plain language to describe the opposite of benefits as “harms.” To emphasize that an individual might or might not experience specific benefits or harms, this report sometimes describes them as “potential.” Identifying a “harm” does not mean that it occurs frequently; harms associated with vaccines are rare. For example, vaccine-associated paralytic polio is an established harm of the oral polio vaccine (OPV), but it is estimated to occur at a rate of 1 in 2.7 million first doses of OPV (WHO, 2023). The committee used different types of evidence to draw conclusions concerning possible associations between vaccination and potential harms. Some study types were not available or were considered uninformative for certain outcomes. Conclusions about causality were informed by the totality of the evidence without applying arbitrary rules or thresholds regarding the number or types of studies required to draw conclusions. For each outcome, the committee discussed the totality of the evidence and used consensus methods to draw conclusions about causality. Iterative discussions about the evidence were particularly important given the committee’s decision not to use a formal grading system for each published article or for the causality conclusions. The committee used expert judgment based on clinical and research expertise and analysis, paying careful attention to ensure that all outcomes under study were evaluated similarly to ensure a consistent approach to the causal conclusions was maintained. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 4 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs The committee adopted the wording for the categories of causal conclusions used by the IOM vaccine safety committees (IOM, 1991, 1994, 2012), and approached the evaluation of evidence from a position of neutrality, presuming neither causation nor lack of causation. The conclusion categories are necessarily asymmetrical: although evidence can establish a causal relationship, the committee determined that it was unlikely that it could establish the absence of one for any harm. Similar to other evidence-review efforts, the committee incorporated the potential role of future research in determining the appropriate conclusion, as described below. See Box S-2 for a description of the categories. CONCLUSIONS Given that this review occurred shortly after vaccines were available, the information in this report is a snapshot in time. New vaccines will be developed, and more research conducted. For example, the evidence does not address the real-world use of the COVID-19 vaccines in which many individuals received a “mix and match” sequence of them. Many people vaccinated for COVID-19 received other vaccines (e.g., influenza) simultaneously. Most of the evidence regarding COVID-19 vaccines was from the primary series; because children were among the last groups to be vaccinated, less evidence exists about them. The committee was not charged to evaluate the benefits of vaccines. All conclusions must be assessed in the context of the established harms of the infections against which a vaccine is directed and the well-documented benefits of vaccines in preventing those harms. The committee makes 85 conclusions in eight chapters about the causal relationship between vaccines and possible harms. Although the committee lacked evidence to establish, accept, or reject a causal relationship for many possible harms, it identified sufficient evidence for 20 conclusions. It is not surprising that evidence is insufficient for the majority; many of the conditions had relatively few studies in the literature from which to draw conclusions. As Box S- 2 indicates, the committee incorporated the notion that further research might lead to a different conclusion for all but conclusions establishing causation. See Tables S-1 and S-2 for all committee conclusions. BOX S-2 Categories of Causation ● Evidence establishes a causal relationship—The totality of the evidence suggests that vaccination can cause this harm. Further research is unlikely to lead to a different conclusion. ● Evidence favors acceptance of a causal relationship—The totality of the evidence suggests that vaccination might cause this harm, but meaningful uncertainty remains. Studies that better minimize bias and confounding, and studies that estimate effects more precisely, could lead to a different conclusion. ● Evidence is inadequate to accept or reject a causal relationship—The available evidence is too limited (e.g., few studies in humans, biased, imprecise) or inconsistent to draw meaningful conclusions in support of or against causality. Future research could lead to a different conclusion. This conclusion also applies to situations in which no studies were identified. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. SUMMARY 5 PREPUBLICATION COPY—Uncorrected Proofs ● Evidence favors rejection of a causal relationship—The totality of the evidence suggests that vaccination does not cause this harm, but meaningful uncertainty remains. The committee acknowledges that individual causal effects are difficult to ascertain and the limitations of applying population average effects to draw conclusions about the causes of specific events in individual people. For example, it is possible that both vaccination and disease cause certain harms. Thus, (1) an event could be more common in an unvaccinated than a vaccinated population and (2) some of the events in the vaccinated population could be caused by vaccination. Research demonstrating a clear mechanism of action, or research demonstrating increased risk among vaccinated people compared with unvaccinated people, could lead to a different conclusion. Conclusions by Vaccine Most of the evidence the committee reviewed addressed BNT162b2.2 This is not surprising, as it was the first available in the United States and many other countries; mRNA- 1273 3 followed quickly, and many studies addressed it as well. Conversely, NVX-CoV2373 was the last vaccine available in the United States, and the committee identified no published studies relevant for review. The U.S. FDA revoked the authorization for Ad26.COV2.S,4 and the small number of studies reflected that short availability. The causality conclusions for the two messenger ribonucleic acid (mRNA) vaccines (BNT162b2 and mRNA-1273) were almost identical; the committee found convincing evidence that established a causal relationship with myocarditis. In contrast, the committee concluded that the evidence favored rejection of a causal relationship between both mRNA vaccines and thrombosis with thrombocytopenia syndrome (TTS), infertility, Guillain-Barré syndrome (GBS), Bell’s palsy (BP), and myocardial infarction (MI). The committee identified numerous studies supporting the conclusions about GBS, BP, and MI. The evidence for TTS and infertility was more limited but still suggestive of no effect. The committee also concluded that the evidence favored rejection of a causal relationship between BNT162b2 and ischemic stroke, but the evidence was inadequate to accept or reject a causal relationship between mRNA-1273 and ischemic stroke, as the data were more limited. Despite the limited use of Ad26.COV2.S in the United States and therefore the limited number of published studies, the committee identified sufficient evidence to conclude that it favored acceptance of a causal relationship with two specific harms, TTS and GBS. The evidence base for these two conclusions were very different. The conclusion about TTS relied on strong mechanistic evidence of binding of vaccine-generated anti-PF4 antibody to platelets in people who developed TTS who had been given ChAdOx1-S, which is a similar platform to Ad26.CoV2.S. Although the mechanistic findings for ChAdOx1-S were stronger, the similar findings with Ad26.COV2.S combined with pharmacovigilance data led the committee to conclude that the evidence favors acceptance of a causal relationship. The conclusion for GBS was based on strong epidemiological studies and pharmacovigilance data. Tables S-1 and S-2 contain the causality conclusions for each potential harm. 2 Refers to the COVID-19 vaccine manufactured by Pfizer-BioNTech under the name Comirnaty®. 3 Refers to the COVID-19 vaccine manufactured by Moderna under the name Spikevax®. 4 Refers to the COVID-19 vaccine manufactured by Janssen. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 6 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs Conclusions by Causal Category The committee made six conclusions that the evidence establishes a causal relationship with vaccination. The evidence for these conclusions fell into two broad categories. The conclusions regarding myocarditis and the mRNA platform–based vaccines, BNT162b2 and mRNA-1273, relied upon extensive data from many sources and well-supported mechanistic evidence. In patients with vaccine-associated myocarditis, elevated levels of spike protein were detected in their blood and on myocardial tissue. Studies in animal models and ex vivo human samples show a connection between myocarditis and the activation of specific immune pathways, such as TLR4/inflammasome/IL-1β, triggered by mRNA COVID-19 vaccines. The conclusions regarding certain shoulder injuries after intramuscular injection (independent of type of vaccine) relied heavily on numerous well-documented case reports and a good mechanistic understanding that injection directly into certain areas of the shoulder could lead to injury of the bursa, tendon, bone, or nerve. The committee also made two conclusions that the evidence favors acceptance of a causal relationship between Ad26.COV2.S and GBS and TTS. The evidence for these two conclusions varied quite a bit, with mechanistic data and pharmacovigilance providing the support for TTS and epidemiological studies for GBS. The committee made conclusions favoring rejection of causality for 12 possible harms. For both GBS and TTS, the committee concluded that the evidence favored rejection with both mRNA platform vaccines but convincingly supported a causal relationship with Ad26.COV2.S. This supports the understanding that the platform distinctly influenced the adverse response. The committee also favored rejection of a causal relationship for the mRNA vaccines and several other outcomes: female infertility, BP, MI, and ischemic stroke (BNT162b2 only). The evidence varied widely for these conclusions. The committee also concluded that the evidence favors rejection of a causal relationship between vaccine injection and chronic rotator cuff disease. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. SUMMARY 7 PREPUBLICATION COPY—Uncorrected Proofs TABLE S-1 Causal Conclusions Regarding COVID-19 Vaccines Chapter Potential Harm Causality Conclusions BNT162b2 (Pfizer-BioNTech) mRNA-1273 (Moderna) Ad26.COV2.S (Janssen) NVX-CoV2373 (Novavax) 3 Guillain-Barré syndrome Favors rejection of a causal relationship Favors rejection of a causal relationship Favors acceptance of a causal relationship I Chronic inflammatory demyelinating polyneuropathy I I I I Bell’s palsy Favors rejection of a causal relationship Favors rejection of a causal relationship I I Transverse myelitis I I I I Chronic headache I I I I Postural orthostatic tachycardia syndrome I I I I 4 Sensorineural hearing loss I I I I Tinnitus I I I I 5 Thrombosis with thrombocytopenia syndrome Favors rejection of a causal relationship Favors rejection of a causal relationship Favors acceptance of a causal relationship I Immune thrombocytopenic purpura I I I I Capillary leak syndrome I I I I Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 8 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs TABLE S-1 Continued Chapter Potential Harm Causality Conclusions BNT162b2 (Pfizer-BioNTech) mRNA-1273 (Moderna) Ad26.COV2.S (Janssen) NVX-CoV2373 (Novavax) 6 Myocardial infarction Favors rejection of a causal relationship Favors rejection of a causal relationship I I Ischemic stroke Favors rejection of a causal relationship I I I Hemorrhagic stroke I I I I Deep vein thrombosis, pulmonary embolism, venous thromboembolism I I I I 7 Myocarditis Establishes a causal relationship Establishes a causal relationship I I Pericarditis without myocarditis I I I I 8 Sudden death I I I I 9 Female infertility Favors rejection of a causal relationship Favors rejection of a causal relationship I I *NOTE: “I” indicates that the evidence was inadequate to accept or reject a causal relationship. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. SUMMARY 9 PREPUBLICATION COPY—Uncorrected Proofs TABLE S-2 Conclusions Regarding Shoulder Injuries After Any Vaccination Specific Shoulder Injury (Chapter 10) Causality Conclusion Subacromial/subdeltoid bursitis caused by direct injection into the bursa Establishes a causal relationship Acute rotator cuff or acute biceps tendinopathy caused by direct injection into or adjacent to the tendon Establishes a causal relationship Chronic rotator cuff disease Favors rejection of a causal relationship Adhesive capsulitis I Septic arthritis I Bone injury caused by direct injection into or adjacent to the bone Establishes a causal relationship Axillary or radial nerve injury caused by direct injection into or adjacent to the nerve Establishes a causal relationship Parsonage-Turner syndrome I Complex regional pain syndrome I NOTE: “I” indicates that the evidence was inadequate to accept or reject a causal relationship. Evidence in Children As described in Chapter 1, vaccine-associated harms may differ in children and adults. For this reason, the committee conducted an in-depth review of the literature on potential harms and COVID-19 vaccines specifically in children (individuals younger than 18). At the time of the review, data on possible harms in children were available only for BNT162b2 and mRNA-1273. Emergency use authorization of COVID-19 vaccines for children occurred later than for adults, and decreased uptake in children, particularly those under 11, led to far less data on possible harms from COVID-19 vaccines in children being available in the literature. CONCLUDING REMARKS The COVID-19 pandemic resulted in a voluminous increase in research for many disciplines on many topics in very little time. Many factors complicated this research. Many investigators and clinicians were treating patients under very challenging circumstances while also conducting research. Vaccines were approved or authorized for use at different times for different populations in different countries. Priority groups among the first vaccines were older people and those with comorbidities that could have put them at risk for adverse events after vaccination. The communities being vaccinated had widespread SARS-CoV-2 infection, so that few studies were able to exclude patients with an infection that occurred simultaneously with vaccination. Thus, some of the outcomes observed after vaccination might reflect harms from infection instead. Patterns of non-SARS-CoV-2 infections changed dramatically during the early days of the pandemic due in part to social distancing and other public health interventions. See the discussion on GBS in Chapter 3 as an example. This complicates the use of historical controls in some studies. Many publications report surveillance findings, which do not use Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 10 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs control populations. Rather, comparisons are made to historical trends, which are not a true contemporaneous unvaccinated population. Other methodologic limitations across many of the studies include challenges in confirming vaccine receipt and diagnostic validity. Many studies in this report were not initiated to support causal inference reviews. Thus, although a particular paper might have had limited utility to this committee, it likely has relevance and immense purpose for others. The committee appreciates the vast amount of work of researchers and clinicians during the pandemic and the contributions of the participants involved in these studies and hopes that the information and conclusions in this report are useful to vaccine researchers and the public health community at large. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. SUMMARY 11 PREPUBLICATION COPY—Uncorrected Proofs REFERENCES CDC (Centers for Disease Control and Prevention). 2023. COVID-19 vaccine effectiveness update. https://covid.cdc.gov/covid-data-tracker/#vaccine-effectiveness (accessed March 8, 2024). FDA (Food and Drug Administration). 2023. Re: Revocation of EUA 27205 - Janssen COVID-19 vaccine. https://www.fda.gov/media/169003/download?attachment (accessed March 1, 2024). Grimes, R. 2023. Overview of injury compensation programs—NASEM committee to review relevant literature regarding adverse events associated with vaccines: Division of Injury Compensation Programs. HRSA (Health Resources and Services Administration). 2023a. Comparison of Countermeasures Injury Compensation Program (CICP) to the National Vaccine Injury Compensation Program (VICP). https://www.hrsa.gov/cicp/cicp-vicp (accessed December 11, 2023). HRSA. 2023b. Countermeasures Injury Compensation Program: Covered countermeasures. https://www.hrsa.gov/cicp/covered-countermeasures (accessed December 20, 2023). IOM (Institute of Medicine). 1991. Adverse effects of pertussis and rubella vaccines. Edited by C. P. Howson, C. J. Howe, and H. V. Fineberg. Washington, DC: National Academy Press. IOM. 1994. Adverse events associated with childhood vaccines: Evidence bearing on causality. Edited by K. R. Stratton, C. J. Howe, and R. B. Johnston, Jr. Washington, DC: National Academy Press. IOM. 2011. Finding what works in health care: Standards for systematic reviews. Edited by J. Eden, L. Levit, A. Berg, and S. Morton. Washington, DC: The National Academies Press. Junqueira, D. R., L. Zorzela, S. Golder, Y. Loke, J. J. Gagnier, S. A. Julious, T. Li, E. Mayo-Wilson, B. Pham, R. Phillips, P. Santaguida, R. W. Scherer, P. C. Gøtzsche, D. Moher, J. P. A. Ioannidis, and S. Vohra. 2023. Consort harms 2022 statement, explanation, and elaboration: Updated guideline for the reporting of harms in randomised trials. British Journal of Medicine. 381:e073725. https://doi.org/10.1136/bmj-2022-073725. Watson, O. J., G. Barnsley, J. Toor, A. B. Hogan, P. Winskill, and A. C. Ghani. 2022. Global impact of the first year of COVID-19 vaccination: A mathematical modelling study. Lancet Infectious Diseases 22(9):1293–1302. https://doi.org/10.1016/S1473-3099(22)00320-6. WHO (World Health Organization). 2023. Polio: Global eradication initiative. https://cdn.who.int/media/docs/default-source/Documents/gpei-cvdpv-factsheet-march- 2017.pdf?sfvrsn=1ceef4af_0 (accessed December 17, 2023). Zorzela, L., Y. Loke, J. P. A. Ioannidis, S. Golder, P. Santaguida, D. Altman, D. Moher, S. Vohra, and PRISMA Harms Group. 2016. PRISMA harms checklist: Improving harms reporting in systematic reviews. British Journal of Medicine. 352:i157. https://doi.org/10.1136/bmj.i157. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 12 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREPUBLICATION COPY—Uncorrected Proofs 1 Introduction Vaccines are a major public health success story, preventing or mitigating the effects of a myriad of infectious diseases. In 1986, the United States faced a problem with vaccine development and production. The threat of litigation over safety concerns related to the whole cell pertussis vaccines in particular led manufacturers to slow research and development and leave the market. Congress addressed what many considered to be a looming crisis for public health by passing the National Childhood Vaccine Injury Act (NCVIA) (P.L. 99-660) to improve federal coordination of vaccine efforts around research and development and address the concerns of those who asserted that they or their children were injured by vaccines. The Vaccine Injury Compensation Program (VICP), housed in the Health Resources and Services Administration (HRSA) in the Department of Health and Human Services and jointly administered by the Department of Justice, serves as a key policy solution developed by Congress. The program includes vaccines recommended for routine use in children or pregnant women, and anyone who receives a covered vaccine is eligible to apply for compensation. The program is funded by a federal excise tax on covered vaccines; the taxes are held in the Vaccine Injury Trust Fund (HRSA, 2023a). VICP has long depended on the reports from the National Academies of Sciences, Engineering, and Medicine (the National Academies) as an important scientific contribution to its compensation decisions, beginning with two studies mandated by NCVIA (Sections 312 and 313 of Public Law 99-660). IOM (1991, 1994) focused on assessing the causal relationship of CDC-recommended childhood vaccines with specific potential harms. That early work was continued by other National Academies committees reviewing the scientific literature regarding the potential for vaccines to cause harm (IOM, 2002, 2012). The committees did not recommend whether or which harms should be compensated but focused on making conclusions about the causal nature of the vaccines and potential harms after a comprehensive review of biologic, clinical, and epidemiological literature. Compensation decisions remain determined by the intricate processes established by VICP (HRSA, 2023b). See HRSA (2023b) for a description of program administration and the claims process. HRSA also administers the Countermeasures Injury Compensation Program (CICP) to provide compensation for those harms by medical countermeasures, which are vaccines, medications, devices, or other preventions, diagnostics, or treatments for a public health emergency or security threat. Established by the Public Readiness and Emergency Preparedness Act of 2005 (P.L. 148, Division C), CICP differs significantly from VICP (HRSA, 2023c). On January 31, 2020, the Secretary of Health and Human Services declared a Public Health Emergency related to SARS-CoV-2 under Section 319 of the Public Health Service Act. The public health emergency expired on May 11, 2023. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 14 VACCINE EVIDENCE REVIEW The public health emergency was declared because SARS-CoV-2 and the disease caused by SARS-CoV-2, COVID-19, were the greatest public health crisis to date of the 21st century. As of February 2024, it had led to an estimated 7 million deaths worldwide, including 1.2 million deaths in the United States (WHO, 2024). COVID-19 was a major cause of death and illness in both adults and children. Long COVID is a particular concern. In 2021, COVID-19 was the third most common cause of death in adults in the United States (CDC, 2021), and from 2020-2022, COVID-19 was among the top 10 causes of death in children in the United States (Flaxman et al., 2023). Part of the public health emergency was the announcement of “Operation Warp Speed,” a rapid response by the federal government to speed vaccine development (for detailed information, see GAO, 2021). Four vaccines were developed and used in the United States, all under Emergency Use Authorization (EUA) (see FDA, 2023), with some now fully approved by the Food and Drug Administration (FDA). However, as of June 1, 2023, FDA revoked the EUA from Ad26.CoV2.S for safety concerns (FDA, 2023b). EUA allowed vaccines to be used before all phase 3 trials were completed.1 COVID-19 vaccines, introduced in 2020, are highly effective in adults and children (CDC, 2023), and were key to control of the pandemic. COVID-19 vaccines are estimated to have prevented 14.4 million deaths worldwide in the first year of vaccination alone (Watson et al, 2022). Although in this report, the committee is tasked with evaluating the causal association with select serious harms, a comparative study analyzing the prevalence and types of side effects following COVID-19 vaccination showed that the most common side effects across different vaccines were flu-like syndrome and local reactions at the injection site, which aligns with the side effect profiles of many vaccines (Yadegarynia et al., 2023). STATEMENT OF TASK HRSA requested that the National Academies convene a committee to review the evidence regarding specific potential harms (see Box 1-1) and the COVID-19 vaccines used in the United States. See Table 1-1 for a list of those vaccines and the naming conventions used in this report. The list of harms to be addressed requested by HRSA are those for which, when the project began, HRSA had claims for compensation. The committee added postural orthostatic tachycardia syndrome (POTS) to its review after presentations at a public meeting. 1 The sentence was updated after the report was shared with the sponsor to clarify the EUA process. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. INTRODUCTION 15 PREPUBLICATION COPY: Uncorrected Proofs BOX 1-1 Statement of Task The National Academies of Sciences, Engineering, and Medicine will convene an ad hoc committee to review the epidemiological, clinical, and biological evidence regarding the relationship between • COVID-19 vaccines and specific adverse events i.e., Guillain-Barré Syndrome (GBS), chronic inflammatory demyelinating polyneuropathy (CIDP), transverse myelitis, Bell’s palsy, hearing loss, tinnitus, chronic headaches, infertility, sudden death, myocarditis/pericarditis, thrombosis with thrombocytopenia syndrome (TTS), immune thrombocytopenic purpura (ITP), thromboembolic events (e.g., cerebrovascular accident (CVA), myocardial infarction (MI), pulmonary embolism, deep vein thrombosis (DVT)), capillary leak syndrome, and • intramuscular administration of vaccines and shoulder injuries. The committee will make conclusions about the causal association between vaccines and specific adverse events. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 16 VACCINE EVIDENCE REVIEW TABLE 1-1 COVID-19 Vaccines Used in the United States Non- Commercial Name Commercial Name Manufacturer Platform Type Adjuvant or Functional Adjuvant U.S. EUA Date U.S. Full Approval Date Approved for Use in BNT162b2 Comirnaty® Pfizer and BioNTech mRNA Self#- LNP and mRNA December 11, 2020 August 23, 2021 Adults and children aged 6+ months mRNA-1273 Spikevax® Moderna mRNA Self# - LNP and mRNA December 18, 2020 January 31, 2022 Adults and children aged 6+ months Ad26.COV2.S* NA Janssen AV Self#- AV February 27, 2021 - Adults (18+) NVX-CoV2373 NA Novavax Protein Subunit Matrix-M® July 13, 2022 - Adults (18+) NOTE: *This vaccine is the same type of platform as ChAdOx1, manufactured by AstraZeneca, but uses a different adenovirus vector. ChAdOx1 is not used in the United States. # mRNA and previously used AV vaccines in the United States do not contain discrete adjuvants. The LNP and AV function as adjuvants to activate the innate immune system. AV: adenovirus vector; EUA: emergency use authorization; mRNA: messenger ribonucleic acid; LNP: lipid nanoparticle. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. INTRODUCTION 17 PREPUBLICATION COPY: Uncorrected Proofs HRSA also requested that the committee review the evidence regarding any vaccine, not specifically COVID-19 vaccines, and shoulder injuries. Claims for compensation for shoulder injuries after vaccination comprise over 63 percent of claims submitted to VICP in fiscal years 2021 and 2022 (Grimes, 2023). The scientific review was requested to help VICP better understand whether vaccination can cause very specific types of shoulder injuries or a more general syndrome that it designated as Shoulder Injuries Related to Vaccine Administration (SIRVA) (HRSA, 2023d). Claims regarding shoulder injuries after routinely administered vaccines are handled by VICP and COVID-19 vaccines by CICP. For the committee’s work, it is irrelevant whether a vaccine is covered under VICP or CICP; National Academies committees do not consider VICP or CICP processes when reviewing the evidence. The committee comprised 15 members with expertise in epidemiology, causal inference, cardiology, rheumatology, gynecology, audiology, neurology, infectious disease, pediatrics, internal medicine, hematology, orthopedics, pharmacoepidemiology, and immunology. Their biosketches can be found in Appendix A. The committee held two sessions open to the public. On January 30, 2023, it heard from representatives of HRSA and CDC on how they intend to use the report and why they asked for the review. On March 30, 2023, the committee held an open session during which members of the public registered to provide 3-minute statements concerning its task. Written material submitted to the committee is in a Public Access File.2 The committee attempted to identify and analyze published literature about the vaccines and potential harms. Although it reviewed the literature thoroughly, it did not conduct what is commonly referred to as a “systematic review,” formal steps of which were described by IOM (2011). The processes and time frame for a systematic review were considered incompatible with this work and, more importantly, the goals of this work were different from those of most systematic reviews and clinical guidelines. The committee was not tasked with estimating the magnitude or strength of associations between vaccinations and outcomes, and the evidence was not expected to be conducive to meta-analysis in any case. To fulfill its narrower goals, the committee did incorporate important attributes of good systematic reviews. A more detailed description of the process by which the committee identified and analyzed the literature follows. The committee does not address the benefits of vaccines. This review addresses evidence only about specific potential harms and vaccines available in the United States. The committee does not make conclusions regarding specific patient cases (such as in published case reports) or whether VICP or CICP should award compensation in individual cases or in general. The committee does aim to present evidence in a way useful to VICP, CICP, claimants and their legal representatives, clinicians, and the public. Vaccines and other medical products can cause both benefits and harms. Harms are sometimes described using terms such as “adverse event,” “adverse effect,” “side effect,” or “safety.” Such terms might not convey the importance of unwanted medical events. Moreover, readers might be confused by the use of different terms with overlapping meanings or the same terms to mean different things in different contexts (Qureshi et al., 2022). For example, “adverse events” are defined in regulatory research as unwanted events not necessarily related to an intervention (e.g., a vaccine, a drug). By comparison, “adverse effects” are both unwanted and related to an intervention. On the other hand, “side effects” might be desirable or unwanted, and they are related to an intervention. Following best practices (Junqueira et al., 2023; Zorzela et al., 2 A list of Public Access File materials can be requested on the study’s National Academies Projects and Activities Repository page: www.nationalacademies.org/our-work/review-of-relevant-literature-regarding-adverse- events-associated-with-vaccines. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 18 VACCINE EVIDENCE REVIEW 2016), this report describes the opposite of benefits as “harms.” To emphasize that an individual patient might or might not experience specific benefits or harms, this report sometimes describes them as “potential.” Identifying a “harm” does not mean that it occurs frequently; harms associated with vaccines are rare. For example, vaccine-associated paralytic polio is an established harm of the oral polio vaccine (OPV), but it is estimated to occur at a rate of 1 in 2.7 million first doses of OPV (WHO, 2023). Literature Search The committee provided the National Academies research librarian with a comprehensive list of search terms for each potential harm. The librarian conducted separate literature searches for epidemiological and mechanistic literature based on the search terms using Embase, Medline, PubMed, Scopus, and Cochrane Central Register of Controlled Trials (Ovid). Epidemiological Evidence Three comprehensive epidemiological literature searches were conducted. Each search included terms specific to each potential harm in at least one search field (i.e., title, abstract, keywords) The list of search terms is available through the project Public Access File.3 The first search was for literature published January 1, 2020–February 28, 2023. Follow- up searches captured literature published February 28–July 7, 2023, and July 7–October 17, 2023. Thus, publications that appeared in the databases after October 17, 2023, are not included in this report. Ad hoc searches were conducted if committee members added a search term and for literature on POTS. The committee restricted its review to U.S. vaccine platforms but included studies conducted outside of the United States. Citations were uploaded to PICO Portal, an online platform used to screen abstracts and full text. Abstracts were reviewed to screen out citations that did not address the potential harm under the committee’s purview and studies that evaluated only vaccine platforms (e.g., inactivated virus vaccine) not approved in the United States. The committee focused its review on original reports and systematic reviews, excluding narrative reviews or commentaries. For systematic reviews, committee members screened each publication and excluded those that were considered unreliable after consideration of the following: no defined criteria for selection of studies, literature search not comprehensive for eligible studies, no assessment of risk of bias in the included studies, and inappropriate methods for meta-analyses (when meta- analyses were reported). Systematic reviews were examined to determine whether they studied the potential harms of interest and for quality of evidence. Committee members evaluated the full text of potentially relevant epidemiological studies and eliminated those that had serious methodologic limitations and were judged unlikely to contribute to the causality assessment. Studies were excluded for reasons such as misclassification of the exposure (vaccination status) and outcomes (e.g., harms were more likely to be recorded in a certain group even if they did not occur more frequently), uncontrolled confounding, selection bias, and substantial missing data (e.g., vaccination status or outcome status is unknown for a large proportion of participants). Misclassification of the exposure means that the specific vaccine was not consistently identified. Misclassification of the outcome means 3 Public Access File materials can be requested by contacting the Public Access Records Office via link on this project’s webpage, www.nationalacademies.org/our-work/review-of-relevant-literature-regarding-adverse-events- associated-with-vaccines. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. INTRODUCTION 19 PREPUBLICATION COPY: Uncorrected Proofs that the potential harms could not be reliably identified. For instance, many studies used diagnosis codes from health care encounters to identify health outcomes—for many outcomes, the codes either are known to perform poorly (e.g., individuals with the code often do not have the outcome, or the code is absent when individuals have experienced the outcome) or have unknown accuracy for validated outcomes. Confounding can occur when an association between vaccination status and the outcome is explained by a common cause that is not completely controlled for in the design and analysis; this is one of the major problems for causal inference using results from observational studies rather than randomized controlled trials (RCTs). Many studies were unable to exclude the possibility of the harms occurring due to SARS-CoV-2 infection. Data extraction was performed on articles that were included at this stage. Pharmacovigilance studies and case reports were identified through the literature search and reviewed if the evidence from the epidemiological studies did not lead the committee to accept or reject a causal relationship. A bibliography of all citations reviewed but not included in this report are available through the project Public Access File.4 Evidence in Children Adverse effects associated with vaccines may differ in children and adults. For this reason, the committee conducted an in-depth review of the literature on potential harms from COVID-19 vaccines specifically in children (those under 18). For context, the vaccines received emergency use authorization (EUA) much later in children than adults, and even later in young children (5–11 years and 6 months to 4 years) than adolescents (12–17 years) (Table 1-2). TABLE 1-2 COVID-19 Vaccine U.S. Food and Drug Administration Emergency Use Authorization Dates, Adults and Children Vaccine Age Group EUA Date BNT162b2 ≥16 years December 11, 2020 12–15 years May 10, 2021 5–11 years October 29, 2021 6 months–4 years June 17, 2022 mRNA-1273 ≥18 years December 18, 2020 6 months–17 years June 17, 2022 NOTES: BNT162b2 refers to the COVID-19 vaccine manufactured by Pfizer-BioNTech under the name Comirnaty®. mRNA-1273 refers to the COVID-19 vaccine manufactured by Moderna under the name Spikevax®. EUA: Emergency Use Authorization. These much later EUA dates and a decrease in SARS-CoV-2 cases after vaccination of adults led to lower immunization rates in children; in May 2023, these were only 13 percent, 39 4 Public Access File materials can be requested by contacting the Public Access Records Office via link on this project’s webpage, www.nationalacademies.org/our-work/review-of-relevant-literature-regarding-adverse-events- associated-with-vaccines. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 20 VACCINE EVIDENCE REVIEW percent, and 68 percent in children aged 6 months to 4 years, 5–11, and 12–17, respectively, according to the Centers for Disease Control and Prevention (AAP, 2023). For these reasons, considerably less data exist on possible harms in children, especially in those under 11, compared to adults. Ad26.COV2.S 5 was never given an EUA for individuals under 18. NVX- CoV2373, although granted an EUA for those aged 12–17 on August 19, 2022, has had very little uptake, so little data exist beyond the original clinical trial on potential harms in children.6 The committee therefore reviewed the available data on COVID-19 vaccines in children, which consisted of data from BNT162b2 7 and mRNA-1273.8 Although there are numerous publications on COVID-19 vaccines in children, the vast majority of these are editorial. commentary or opinion pieces, or case reports or small case series. These publications typically do not provide the quality of evidence needed for evaluation of the relationship of potential harms to vaccine administration. Published data on COVID-19 vaccines in children was reviewed in depth by the committee, and all publications that provided data that could be used to evaluate the relationship of the vaccine to adverse events were included in the analysis. For children, and particularly for children younger than 12 years of age, there was a paucity of data, due to later authorization of COVID-19 vaccines for children and lower immunization rates in children as compared to adults, resulting in less study of adverse events in children than adults. Mechanistic Evidence The committee aimed to understand immune mechanisms of the vaccine platforms potentially related to harms, as described in Chapter 2, by conducting a general search. The first search was limited to studies in humans and identified literature published January 2021–March 2023. A second search looked for information specific to the potential harms under study; it identified literature published January 2000–April 2023 and explored general mechanisms underlying vaccine–immune interactions, focusing on non-SARS-CoV-2 messenger ribonucleic acid (mRNA) and adenovirus-vector (AV) vaccines. A final literature search was conducted in September 2023. Included articles encompassed a broad spectrum of research, including human trials, murine studies, other animal models, computational modeling, and in vitro studies. Ad hoc searches conducted throughout the study were particularly informative as the committee investigated possible mechanisms. The literature search aimed to identify studies elucidating the mechanism underlying specific harms of COVID vaccination and to identify studies quantifying the effect of vaccination on components of the immune system in general. In addition, ad hoc literature searches were performed to review the mechanism of specific harms outside of the vaccination context (e.g., Guillain-Barré syndrome). In the case of shoulder injury, the mechanistic evidence was largely derived from imaging (e.g., MRI) provided in case reports and case series. 5 Refers to the COVID-19 vaccine manufactured by Janssen. 6 Refers to the COVID-19 vaccine manufactured by Novavax. 7 Refers to the COVID-19 vaccine manufactured by Pfizer-BioNTech under the name Comirnaty®. 8 Refers to the COVID-19 vaccine manufactured by Moderna under the name Spikevax®. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. INTRODUCTION 21 PREPUBLICATION COPY: Uncorrected Proofs CAUSALITY ASSESSMENT Types of Evidence The committee used different types of evidence to draw conclusions concerning possible associations between vaccination and harms. Conclusions about causality were informed by the totality of the evidence without applying arbitrary rules or thresholds regarding the number or types of studies required to draw conclusions. Some study types were not available or were considered uninformative for certain outcomes, so the following chapters do not necessarily discuss all the study types described below. The committee reviewed the literature following a well-accepted hierarchy of evidence, beginning with randomized clinical trials and controlled observational epidemiological studies. The committee proceeded to review additional evidence (uncontrolled epidemiological evidence and case reports) until the committee felt it reviewed sufficient and appropriate evidence to support a specific causal conclusion. For example, the committee did not review uncontrolled pharmacovigilance studies and case reports if they felt the observational epidemiological literature was sufficient to support a conclusion or if they felt evidence of those uncontrolled designs were unlikely to contribute to a causal conclusion. The committee notes that uncontrolled studies would likely have been excluded from consideration if they had followed strict inclusion and exclusion criteria, as is done in systematic reviews. However, given the limited information regarding some of the potential harms being reviewed, the committee felt it important to be broad in its consideration of evidence. Clinical Trials For each potential harm, the committee examined evidence in Phase III RCTs, including published results from clinical trials and the documents reviewed and produced by FDA in consideration of the applications by manufacturers for EUA and full approval, when available. RCTs can produce valid causal estimates (e.g., because they minimize selection bias and confounding). Associations detected in RCTs could support causal conclusions, especially for increases in common harms or very large increases in uncommon harms. The committee was aware that RCTs were not designed to assess rare harms, and RCTs did not enroll enough participants to estimate rare events reliably. Some harms are so rare that they would not be expected to occur in RCTs even if they were caused by vaccination. Lack of evidence from RCTs would usually be considered uninformative (rather than evidence of no association). Nonrandomized Studies The committee also considered evidence from nonrandomized studies (controlled observational studies and uncontrolled screening or pharmacovigilance studies) that used appropriate methods to estimate causal effects. Although the committee determined that controlled observational studies were at greater risk of bias compared with RCTs, estimates from studies that minimized bias were considered potentially informative. Notably, positive associations between vaccination and harms could provide evidence of causality. The committee interpreted negative and null findings cautiously. Compared with RCTs, large observational studies might estimate effects with greater precision but greater bias; consequently, it would be difficult to exclude small causal effects based on evidence from nonrandomized studies alone. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 22 VACCINE EVIDENCE REVIEW The committee also considered evidence from pharmacovigilance and surveillance studies, although estimates from these studies were generally considered at greater risk of bias compared with well-designed case-control and cohort studies. Case Reports The committee determined that case reports should inform causal conclusions when temporal and biological relationships between vaccination and harm were readily observable in the reports. In particular, case reports might provide useful evidence about shoulder injuries (Chapter 10). For harms with unclear onset and myriad potential causes, the committee determined that case reports were unlikely to be informative. Mechanisms The committee considered evidence concerning possible mechanisms of action, including findings from human and other studies. Identifying a plausible mechanism could inform the committee’s interpretation of evidence concerning associations in clinical trials and observational studies but not necessarily lead to conclusions favoring causal associations. Because mechanisms might be unknown, lack of mechanistic evidence did not preclude conclusions that vaccination caused harm. Extrapolation The committee considered evidence about each specific vaccine and each harm and discussed whether evidence for some vaccines should inform conclusions about others that used the same platform (e.g., mRNA, adenovirus vector (AV)). For example, mechanistic and clinical evidence establishing a causal relationship between one vaccine and a harm could inform conclusions about the effects of similar vaccines. The committee extrapolated evidence from one vaccine of a specific platform to another vaccine cautiously. In particular, the literature regarding AV ChAdOx1-S (not available in the United States) was considered in assessing TTS risk from Ad26.COV2.S (see Chapter 5). Causal Conclusions Working groups assigned to each outcome performed the initial screen, data abstraction, and evidence review in advance of full committee discussions. Key elements in the data abstraction included study design, sample size, comparison group, risk period, vaccine and outcome ascertainment, and methodological strengths and limitations, including risk of bias considerations. Evidence tables and narratives were presented to the full committee for extensive discussion, including in depth re-examination of individual studies and the preliminary causality conclusion in many circumstances in order to reach a common understanding of the strengths and weaknesses of the evidence and consensus conclusions. This was particularly important when a study was used by more than one working group; a particular research paper might have serious limitations or utility to the committee for one outcomes, but not for every outcomes studied. For each outcome, the committee discussed the totality of the evidence and used consensus methods to draw conclusions about causality. Iterative discussions are particularly important given the committee’s decision not to use a formal grading system for each published article or for the causality conclusions. The committee used expert judgment based on clinical and research expertise and analysis, paying careful attention to ensure that all outcomes under Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. INTRODUCTION 23 PREPUBLICATION COPY: Uncorrected Proofs study were evaluated similarly to ensure a consistent approach to the causal conclusions was maintained. The committee adopted the wording of the causality conclusions developed by National Academies/Institute of Medicine committees and approached the evaluation of evidence from a position of neutrality, presuming neither causation nor lack of causation. The causal conclusion categories are necessarily asymmetrical: although evidence can establish a causal relationship, the committee determined it was unlikely that it could establish the absence of one for any harm. Similar to other evidence-review efforts, the committee incorporated the potential role of future research in determining the appropriate conclusion, as described below. The following are the categories of causation used by the committee: ● Evidence establishes a causal relationship—The totality of the evidence suggests that vaccination can cause this harm. Further research is unlikely to lead to a different conclusion. ● Evidence favors acceptance of a causal relationship—The totality of the evidence suggests that vaccination might cause this harm, but meaningful uncertainty remains. Studies that better minimize bias and confounding, and studies that estimate effects more precisely, could lead to a different conclusion. ● Evidence is inadequate to accept or reject a causal relationship—The available evidence is too limited (e.g., few studies in humans, biased, imprecise) or inconsistent to draw meaningful conclusions in support of or against causality. Future research could lead to a different conclusion. This conclusion also applies to situations in which no studies were identified. ● Evidence favors rejection of a causal relationship—The totality of the evidence suggests that vaccination does not cause this harm, but meaningful uncertainty remains. The committee acknowledges that individual causal effects are difficult to ascertain and the limitations of applying population average effects to draw conclusions about the causes of specific events in individual people. For example, it is possible that both vaccination and disease cause certain harms. Thus, (1) an event could be more common in an unvaccinated population than a vaccinated population and (2) some of the events in the vaccinated population could be caused by vaccination. Research demonstrating a clear mechanism of action, or research demonstrating increased risk among vaccinated people compared with unvaccinated people, could lead to a different conclusion. OUTLINE OF THE REPORT Chapter 2 contains a brief review of the major mechanisms by which vaccines affect the immune system. Chapters 3–9 address the evidence regarding COVID-19 vaccines and the specific outcomes listed in the Statement of Task. The structure of the chapters is similar but not identical. Chapters other than Chapters 8 (Sudden Death) and 9 (Female Infertility) contain conclusions about more than one outcome. Each outcome is addressed separately. Each outcome- specific section begins with a description of the outcome under review. A brief description of pathophysiologic mechanisms and the possible role of COVID-19 vaccines follows. The epidemiologic evidence section contains the evidence the committee depended upon in reaching a causal conclusion. Evidence that did not contribute is not described. The most influential Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 24 VACCINE EVIDENCE REVIEW evidence is portrayed in detail in tables within each section and described briefly in the text. Each section ends with a summary of the most compelling argument in support of the conclusion and the section ends with the causal conclusion. Chapter 10 reviews of the shoulder injuries after intramuscular administration of any vaccine, not limited to COVID-19 vaccines. The report ends with crosscutting summaries of the evidence in Chapter 11. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. INTRODUCTION 25 PREPUBLICATION COPY: Uncorrected Proofs REFERENCES AAP (American Academy of Pediatrics). 2023. Summary of data publicly reported by the Centers for Disease Control and Prevention. https://www.aap.org/en/pages/2019-novel-coronavirus-covid- 19-infections/children-and-covid-19-vaccination-trends (accessed December 12, 2023). CDC (Centers for Disease Control and Prevention). 2021. Leading causes of death https://www.cdc.gov/nchs/fastats/leading-causes-of-death.htm (accessed March 6, 2024). CDC. 2023. 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Washington, DC: The National Academies Press. Junqueira, D. R., L. Zorzela, S. Golder, Y. Loke, J. J. Gagnier, S. A. Julious, T. Li, E. Mayo-Wilson, B. Pham, R. Phillips, P. Santaguida, R. W. Scherer, P. C. Gøtzsche, D. Moher, J. P. A. Ioannidis, and S. Vohra. 2023. Consort harms 2022 statement, explanation, and elaboration: Updated guideline for the reporting of harms in randomised trials. British Journal of Medicine 381:e073725. https://doi.org/10.1136/bmj-2022-073725. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 26 VACCINE EVIDENCE REVIEW NASEM (National Academies of Sciences, Engineering, and Medicine). 2023. Review of relevant literature regarding adverse events associated with vaccines. https://www.nationalacademies.org/our-work/review-of-relevant-literature-regarding-adverse- events-associated-with-vaccines (accessed December 18, 2023). Qureshi, R., E. Mayo-Wilson, and T. Li. 2022. Harms in systematic reviews paper 1: An introduction to research on harms. Journal of Clinical Epidemiology 143:186–196. https://doi.org/10.1016/j.jclinepi.2021.10.023. WHO (World Health Organization). 2024. Number of COVID-19 deaths reported to WHO. https://data.who.int/dashboards/covid19/deaths?n=c (accessed March 6, 2024). Watson, O. J., G. Barnsley, J. Toor, A. B. Hogan, P. Winskill, and A. C. Ghani. 2022. Global impact of the first year of COVID-19 vaccination: A mathematical modelling study. Lancet Infectious Diseases 22(9):1293–1302. https://doi.org/10.1016/S1473-3099(22)00320-6. Yadegarynia, D., S. Tehrani, F. Hadavand, S. Arshi, Z. Abtahian, A. Keyvanfar, A. Darvishi, A. Zarghi, L. Gachkar, I. A. Darazam, and M. Farahbakhsh. 2023. Side effects after COVID-19 vaccination: A comparison between the most common available vaccines in Iran. Iranian Journal of Microbiology 15(2):189–195. https://doi.org/10.18502/ijm.v15i2.12467. Zorzela, L., Y. Loke, J. P. A. Ioannidis, S. Golder, P. Santaguida, D. Altman, D. Moher, S. Vohra, and P. h. group. 2016. PRISMA harms checklist: Improving harms reporting in systematic reviews. British Journal of Medicine 352:i157. https://doi.org/10.1136/bmj.i15. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREPUBLICATION COPY: Uncorrected Proofs 2 Immunologic Response to COVID-19 Vaccines The global pandemic stemming from the emergence of the novel SARS-CoV-2 virus in late 2019 made it critical to develop efficacious vaccines. This public health crisis initiated global efforts to produce vaccines to reduce viral transmission and protecting individuals from life-threatening infections (Diamond and Pierson, 2020). Several COVID-19 vaccines were rapidly developed using a variety of platforms. Concomitant with the release of vaccines, concerns arose around vaccine-induced harms. To better understand how vaccine mediated harms may arise, it is important to know how specific COVID-19 vaccines initiate an immune response. Charged with examining biological mechanisms, the committee conducted a comprehensive review of the current literature, examining the available evidence encompassing clinical trials, epidemiology studies, case reports, preclinical and translational in vitro or in silico studies, and insights gained from animal models. The committee analyzed a diverse array of vaccine-mediated harms and a variety of vaccine platforms and compiled a list of mechanisms that were deemed most plausible in contributing to the emergence of vaccine-mediated adverse reactions following COVID-19 vaccination. Throughout these deliberations, the committee engaged in in-depth discussions regarding the pathophysiology that may be involved and the requisite evidentiary support necessary to establish the presence of a particular mechanism. FUNDAMENTALS OF THE IMMUNE RESPONSE The human immune response is initiated by the innate immune system which activates the adaptive immune system. Both the innate and adaptive arms of the immune response play a pivotal role in combating pathogens, such as SARS-CoV-2, and establishing long-term immunity. They are also both important in producing an effective immune response and long- term immunity (immunological memory) after vaccination. The innate immune system is the “first responder” to foreign agents, such as viral infections or physical tissue damage. It comprises physical defenses, such as the skin, and cellular components, such as macrophages, mast cells, dendritic cells, neutrophils, and natural killer cells. The innate immune response is not pathogen specific at the single amino acid (AA)/protein epitope level (i.e., antigen) but recognizes categories of pathogens, such as viruses, bacteria, parasites, and tissue damage, based on molecular patterns that are specific to particular microbes (Chaplin, 2010). A key element of this pathogen recognition system are pattern recognition receptors (PRRs), with Toll-like receptors (TLRs) being a notable subgroup. For example, TLR3 is involved in canonically recognizing double-stranded RNA, commonly associated with viral infections, however, evidence of TLR3 recognition of single-stranded RNA vaccines has been shown (Teijaro and Farber, 2021). TLR4, which recognizes Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 28 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs lipopolysaccharides from Gram-negative bacteria, some viral infections, and self-like ATP from damaged mitochondria, may play a role in responses to messenger ribonucleic acid (mRNA) vaccines, which contain mRNA within lipid nanoparticles (LNPs) that augment innate immune responses. TLR7 and TLR8, recognizing single-stranded RNA, are integral to the immune response against RNA-based vaccines, such as certain COVID-19 vaccines. Meanwhile, TLR9, which detects unmethylated CpG motifs in bacterial and viral DNA, is used in some vaccines as an adjuvant (a substance in vaccines that enhances the immunological response to the antigen). The activation of these TLRs triggers signaling pathways that lead to cytokine and type I interferon production, crucial for initiating adaptive immune responses (Fitzgerald and Kagan, 2020). After activation by the innate immune system, the adaptive immune system develops an antigen-specific immune response to a specific pathogen that is based on particular amino acids (AA)/protein sequences (antigens). Macrophages and dendritic cells are fundamental in presenting antigens to adaptive immune cells, initiating an antigen-specific response crucial for establishing long-lasting immunological memory. Because of the high specificity of the adaptive immune response, it can distinguish not only a specific virus but also a specific strain of that virus. Memory occurs primarily at the T cell and B cell levels. B cells develop into plasma cells that release antigen-specific antibodies that are critical for rapidly clearing infections when they are next encountered the next time. T cells, on the other hand, play a crucial role in immune memory by recognizing and responding to previously encountered antigens, aiding in the rapid mobilization of the immune system during subsequent infections. The development of strong antigen-specific T cell and B cell-antibody memory is a primary goal of vaccine development. All types of vaccines strongly stimulate an innate immune response to direct the adaptive immune response to make protective antigen-specific T and B cells and antibody responses against the target infection. COVID-19 vaccines (Figure 2-1), including traditional protein-based vaccines (NVX-CoV2373 1), mRNA vaccines (e.g., BNT162b2 2 and mRNA-1273 3) and adenovirus-vector (AV) vaccines (e.g., Ad26.COV2.S 4 and ChAdOx-2/nCoV-19 5), are engineered to stimulate both innate and adaptive immune responses. The mRNA vaccines deliver genetic material coding for the SARS-CoV-2 spike S-protein into host cells (Martinez-Flores et al., 2021) so that an antigen-specific adaptive immune response will be generated against it. The mRNA vaccine may be able to activate resident innate immune cells at the injection site, but it primarily takes effect after the spike protein is generated within cells (Verbeke et al., 2022). The mRNA strands are structurally optimized to prevent degradation by incorporating pseudouridines (Kim et al., 2022) and mRNA into lipid nanoparticles (Ndeupen et al., 2021), which both further protects the RNA transcript from degradation and facilitates cell entry (Pardi et al., 2015). Certain components within the LNP layer may also act as adjuvants by activating TLRs on antigen presenting cells and the innate immune response to induce an enhanced adaptive immune response against the spike protein (Alameh et al., 2021). Protein-based vaccines often require an adjuvant to stimulate the innate immune response; AV vaccines have an innate immune- activating ability because they are viral vectors. 1 Refers to the COVID-19 vaccine manufactures by Novavax. 2 Refers to the COVID-19 vaccine manufactured by Pfizer-BioNTech under the name Comirnaty®. 3 Refers to the COVID-19 vaccine manufactured by Moderna under the name Spikevax®. 4 Refers to the COVID-19 vaccine manufactured by Janssen. 5 Refers to the COVID-19 vaccine manufactured by Oxford-AstraZeneca. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 29 PREPUBLICATION COPY: Uncorrected Proofs Innate antigen presenting cells, particularly mast cells, macrophages, and dendritic cells, are instrumental in activating an adaptive immune response. They capture, process, and present pathogen-specific antigens to T cells, inducing a highly targeted adaptive response and immunological memory. Dendritic cells are particularly important in stimulating adaptive immune responses from the draining lymph nodes while resident mast cells and macrophages play key roles at tissue sites. In the milieu of COVID-19 vaccines, antigen presenting cells are vital for identifying and presenting the vaccine-derived spike protein to helper T cells, thereby producing spike protein–specific T and B cells. T cells, comprising helper T cells (CD4+) and cytotoxic T cells (CD8+), play multifaceted effector roles during an infection such as SARS-CoV-2. Helper T cells facilitate B cell activation and enhance the function of cytotoxic T cells, which directly attack and destroy virally infected cells. The adaptive immune cell memory induced by COVID-19 vaccines ensures a rapid antigen-specific T and B cell/antibody response when the vaccinee encounters SARS-CoV-2 in the future. SARS-COV-2 AND VACCINE TARGET OF THE SPIKE PROTEIN SARS-CoV-2 is characterized by several structural proteins; the spike (S) glycoprotein and the nucleocapsid (N) protein are primary targets for the immune response (Krammer, 2020). The spike protein is a major virus surface protein crucial for viral entry into host cells; it binds to the angiotensin-converting enzyme 2 (ACE2) receptor on host cells (Walls et al., 2020). Structurally, the spike protein is a class I viral fusion glycoprotein comprising of two subunits: the S1 subunit, responsible for receptor binding, and the S2 subunit, involved in fusion. These subunits are connected by a furin cleavage site, unique to SARS-CoV-2 (rather than all SARS viruses), and the protein is cleaved post translationally at this furin cleavage site. The receptor- binding domain (RBD) within the S1 subunit is particularly critical for viral entry to cells, as it directly interacts with the ACE2 receptor, initiating conformational changes leading to membrane fusion and viral entry (Kirchdoerfer et al., 2016; Wrapp et al., 2020). In addition, the spike protein is the only SARS-CoV-2 antigen recognized to stimulate neutralizing antibodies (Xiaojie et al., 2020). A number of other receptors are important in viral entry but not described in this report. The spike protein has been the primary focus in vaccine developments due to its essential role in viral entry to host cells. Vaccines contain (subunit vaccines, such as NVX-CoV2373) or generate production of (mRNA and AV vaccines) the spike protein to elicit an immune response in the absence of infection. Typically, adjuvants are also needed to induce a strong immune response because the antigen itself (without an active infection) does not do so in individuals who have not encountered SARS-CoV-2. The goal of all vaccine platforms is to contain or produce a stable form of the S protein that will not degrade or be cleared from the body without activating the immune response. Two main mRNA vaccine strategies have been employed to stabilize the spike protein in its prefusion conformation, which is essential for preserving epitopes that are sensitive to degradation. One method, used in both mRNA-1273 and BNT162b2 vaccines, introduces mutations in the mRNA transcript (proline substitutions at positions 986 and 987), which maintain the spike glycoprotein in the prefusion state (Pallesen et al., 2017; Wrapp et al., 2020). Another strategy, not employed by the current vaccines, involves designing an mRNA construct Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 30 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs where the full-length spike protein lacks the furin cleavage site (∆furin), preventing posttranslational cleavage (Laczko et al., 2020; Lederer et al., 2020). As an alternative to targeting the full-length spike protein, some vaccines focus solely on RBD (Bettini and Locci, 2021), which contains multiple epitopes that can be effective targets for virus neutralization, making it a potent target for vaccine strategies (Robbiani et al., 2020; Zost et al., 2020). For instance, BNT162b1 vaccine candidate developed by BioNTech/Pfizer encodes a secreted trimerized version of RBD. The choice of the full-length spike protein or smaller RBD of the spike protein in vaccine design balances the benefits of eliciting a broader immune response with the full-length protein versus focusing on the highly neutralizing epitopes in the RBD. However, due to its favorable immunogenicity to reactogenicity profiles, BNT162b2, encoding full length spike protein, was chosen as the leading vaccine candidate (Khehra et al., 2021). TYPES OF COVID-19 VACCINES Several COVID-19 vaccines have been developed and authorized for use in the United States, using several different vaccine platforms (Figure 2-1). The mRNA vaccines, such as BNT162b2 and mRNA-1273, use LNP-encapsulated mRNA to encode the SARS-CoV-2 spike protein. This technology prompts host cells to produce the spike protein, subsequently eliciting innate and adaptive immune responses and, most importantly, immunological memory. Adenovirus vector vaccines, such as Ad26.COV2.S (emergency use authorization was revoked by FDA on June 1, 2023) and AZD1222 (not used in the United States), employ modified adenoviruses to deliver DNA encoding the spike protein. Protein subunit vaccines, such as NVX- CoV2373, consist of recombinantly produced viral proteins (such as the spike protein or its epitopes) combined with the Matrix-M® adjuvant, which enhances the immunogenicity of the protein antigen, leading to a more robust immune response. Each platform has distinct immunogenic profiles and mechanisms for eliciting an immune response. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 31 PREPUBLICATION COPY: Uncorrected Proofs FIGURE 2-1 COVID-19 vaccines contributing to this report and their mechanism of action. NOTES: (A) mRNA Vaccines: Upon injection, mRNA encapsulated in lipid nanoparticles (LNPs) is delivered into myocytes or bystander cells. The mRNA is released from LNPs and translated by ribosomes to produce the viral antigen, such as the spike protein (S), which is secreted. Antigen- presenting cells (APCs) such as dendritic cells (DCs) uptake the secreted antigen, initiating an immune response. (B) Adenoviral Vector Vaccines: Adenoviral vectors containing viral DNA enter myocytes or bystander cells, where they uncoat. The DNA, containing a nuclear localization signal, is transported to the nucleus and transcribed into mRNA. The extrachromosomal DNA does not integrate into the host genome. The mRNA is translated into protein, which is secreted and uptaken by APCs, initiating an immune response. (C) Subunit Vaccines: Pre-formed viral protein, such as the spike protein (S), is delivered. Antigen-presenting cells, particularly resident dendritic cells (DCs), uptake the protein to initiate an immune response. Additionally, M-matrix adjuvants enhance this response. # Ad26.COV2.S is no longer authorized under EUA in the United States as of June 1, 2023. *ChAdOx1-S is not used in the United States. Created with BioRender.com. mRNA Vaccines The advent of mRNA vaccines has marked a revolutionary leap in the field of immunology and vaccine development, particularly underscored by their critical role in combating the COVID-19 pandemic. These vaccines represent a significant departure from traditional vaccine platforms, providing a number of new advantages, including rapid development, high efficacy and safety, and rapid adaptation to new viral strains (Welsh, 2021). This technology holds promise for preventing serious outcomes and/or spread from viral infections. Developing mRNA vaccines, although conceptually straightforward, involves a complex design process. These vaccines function by delivering mRNA encoding a target antigen, such as the SARS-CoV-2 spike protein, into host cells. Once in the cytoplasm, the cells use their Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 32 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs own machinery to translate the mRNA into the target protein, which is released from the host cell, usually in an extracellular vesicle (Trougakos et al., 2022), activating an innate immune response that also prompts the adaptive immune system to mount a memory response against the spike (S) protein. The next time the individual sees the spike protein during an active infection or vaccine boost, the immune system rapidly mounts a highly protective T and B cell/antibody response. For a detailed depiction of the sequence through which SARS-CoV-2 mRNA vaccines elicit immune responses, from their administration to the priming of T cells and initiation of germinal center reactions, refer to Figure 2-2. FIGURE 2-2 Immune responses to intramuscular administration of SARS-CoV-2 mRNA vaccines. NOTES: Immune responses triggered by SARS-CoV-2 mRNA vaccines involve a sequence of events starting with their intramuscular administration. These vaccines, which include mRNA encapsulated in lipid nanoparticles (mRNA-LNPs) or the antigen they produce, are first taken up by antigen-presenting cells (APCs) such as dendritic cells. After uptake, these APCs migrate to the lymph nodes, where they activate both CD4 and CD8 T lymphocytes. This process of T cell priming and its subsequent steps are discussed comprehensively in scientific literature. Following priming, CD8 T cells may differentiate into cytotoxic T lymphocytes capable of destroying virus-infected cells, while CD4 T cells may evolve into either Th1 cells or T follicular helper (Tfh) cells. Tfh cells are pivotal in initiating the germinal center reaction, a critical process for the development of high-affinity memory B cells and long-lived plasma cells that secrete antibodies. The direction of Tfh cell differentiation towards a Th1 or Th2 phenotype influences the isotype of antibodies produced by these plasma cells, affecting the body's immune response to the vaccine. SOURCE: Bettini and Locci, 2021. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 33 PREPUBLICATION COPY: Uncorrected Proofs One of the initial challenges of mRNA vaccines was the inherent nature of unmodified mRNA, which is extremely labile and highly immunogenic, making it unsuitable for direct use in vaccines (Pardi et al., 2018). Karikó et al. tested various modifications to nucleosides in mRNA molecules (Kariko et al., 2008). They tested modifications, such as pseudouridine, 5- methylcytidine, N6-methyladenosine, 5-methyluridine, and 2-thiouridine. The substitution of uridine with N1-methyl-pseudouridine (m1Ψ) led to a tenfold increase in translation efficiency compared to unmodified mRNA (Karikó et al., 2008). Moreover, mRNA with this modification was not recognized by the pathogen-associated molecular pattern (PAMP) sensing mechanisms, such as TLRs or retinoic acid-inducible gene I (RIG-I), thus avoiding excessive inflammation, RNA degradation, and potential harms (Karikó et al., 2008; Pardi et al., 2018). This m1Ψ modification has been adopted in the design of several mRNA vaccine candidates, including the widely used mRNA-1273 and BNT162b2 (Corbett et al., 2020; Walsh et al., 2020). Although it is possible to inject naked mRNA directly for immunization, this approach is generally inefficient (Cao and Gao, 2021). For the mRNA to be translated into proteins in the host cell, it must penetrate the cell’s lipid membrane to reach the cellular ribosomes. To facilitate efficient protein translation, delivery methods that ensure the cytosolic localization of mRNA are essential. Although standard laboratory lipid encapsulation methods, such as lipofectamine, were effective in vitro, they were cytotoxic and less efficient in vivo (Cao and Gao, 2021; Karikó et al., 2008). The encapsulation of mRNA into LNPs significantly contributes to its stability and uptake by the cells; LNPs effectively transport mRNA within the body and, upon intramuscular injection, can be taken up by antigen-presenting cells at the injection site and in nearby lymph nodes, facilitating both innate and adaptive immune responses. Furthermore, LNPs provide protection against nuclease-mediated degradation of the mRNA. The composition of LNPs is often proprietary, but they are known to contain a mixture of ionizable cationic lipids, cholesterol, phospholipids, and polyethylene glycols (PEGs), which self-assemble into nanoparticles of approximately 100 nanometers in diameter to encapsulate the mRNA (Cullis and Hope, 2017; Maier et al., 2013). Many of these components are known to be immunogenic and can act as adjuvants to stimulate the innate immune response to the spike protein. In fact, the composition of the LNP can be tailored to enhance the immune system's response to the vaccine by inducing robust Tfh cell and humoral responses, making LNPs not only a delivery vehicle but also an adjuvant-like component of mRNA vaccines (Alameh et al., 2021). Adenovirus Vector Vaccines Adenovirus vector-based vaccines (AV) have emerged as a key player in COVID-19 vaccine development, leveraging the unique properties of adenoviruses. These linear double- stranded DNA viruses, typically responsible for respiratory infections in children and adults, possess stable genes and efficient transduction capabilities (ability to transfer genetic materials), making them ideal vaccine vectors (Lukashev and Zamyatnin, 2016). Adenoviruses do not integrate into the host genome but remain in a non-genome episomal state; meaning the injected genetic material translocates into the nucleus but does not integrate into the host DNA (Coughlan, 2020; Walsh et al., 2020). This aspect is significant because it mitigates concerns about potential long-term genetic changes in the host’s cells. In some other types of viral vectors, the viral DNA could integrate into the host’s genome, which could lead to unintended genetic alterations (Bulcha et al., 2021). However, with AV vaccines, this risk is greatly reduced because the adenovirus DNA remains separate from the host’s DNA. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 34 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs The adenovirus’s nucleocapsid is composed of fiber, penton, and hexon proteins, contributing to its robustness and versatility as a vector. Over 150 primate adenoviruses have been identified, with many being developed for vaccines due to their cost-effectiveness, thermostability, and ability to induce strong immune responses (Chavda et al., 2023). A significant challenge is pre-existing immunity to common adenovirus serotypes in humans. To circumvent this, rare adenoviruses are employed, such as Ad26 or chimpanzee adenoviruses, which are less likely to be neutralized by pre-existing human antibodies. These vectors have demonstrated effectiveness in both animal models and human studies, despite the varying levels of pre-existing immunity across populations (Ewer et al., 2017; Geisbert et al., 2011). In the context of COVID-19 vaccines, AZD1222, also known as “Covishield” by the Serum Institute of India, uses the ChAdOx1 AV. It carries the gene for the SARS-CoV-2 spike protein (ChAdOx1-S), which is expressed in its trimeric prefusion conformation (Watanabe et al., 2021). Janssen Pharmaceuticals developed Ad26.COV2.S, using an Ad26 vector that encodes the spike protein with specific modifications (K986P and V987P) to enhance immunogenicity by locking the spike in its prefusion conformation (Bos et al., 2020). This vaccine is distinguished by its single-dose regimen. Protein Subunit Vaccines Protein-based vaccines, a well-established class of vaccines, use specific proteins (or protein fragments) from a pathogen to elicit an immune response without introducing the complete pathogen. These vaccines are known for their safety, as they do not contain live components of the pathogen, reducing the risk of vaccine-induced disease, but they are also less immunogenic and require adjuvants or other interventions (Pollard and Bijker, 2021). These vaccines fall into two main categories: subunit vaccines, which include only the parts of the virus that best stimulate the immune system, and toxoid vaccines, which use a toxin produced by the pathogen that has been made harmless but still triggers immunity. Toxoid vaccines include diphtheria and tetanus vaccines, which use inactivated forms of the toxins produced by these bacteria. Subunit vaccines include hepatitis B, which uses a surface protein from the virus, the pertussis toxin component of the DtaP vaccine, and NVX-CoV2373. NVX-CoV2373 comprises recombinantly produced spike proteins combined with Novavax’s proprietary Matrix-M® adjuvant (Keech et al., 2020). The spike protein used in the vaccine is produced by baculovirus expression in Spodoptera frugiperda insect cells, a method known for its ability to yield complex, properly folded proteins (Jarvis, 2003). This strategy ensures that the spike protein maintains its prefusion conformation, which is known to expose critical neutralizing epitopes more effectively than the post-fusion conformation (Bowen et al., 2021; Keech et al., 2020). The adjuvant is a critical component that significantly boosts the innate immune response to the spike protein. It is based on saponin, derived from the Quillaja saponaria tree, and combined with cholesterol and phospholipid to form nanoparticles. These nanoparticles enhance the immune response by stimulating the entry of the antigen into antigen- presenting cells and activating these innate cells. This adjuvant has been shown to boost both the quantity and quality of the immune response, leading to higher levels of neutralizing antibodies and a more robust T cell response (Stertman et al., 2023) to infection. In addition, adjuvants enable the use of smaller amounts of antigen. Producing neutralizing antibodies is the goal for most vaccines, as they bind to a pathogen and block its ability to infect cells, effectively Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 35 PREPUBLICATION COPY: Uncorrected Proofs neutralizing its disease-causing capabilities. In addition to antibody production, the orchestration of a robust T cell response is paramount, as these cells not only assist in the maturation of antibody-producing B cells but also identify and eliminate infected host cells, thereby mitigating the pathogen's proliferation and ensuring a comprehensive immunological defense. This vaccine’s storage and handling requirements are less stringent than those of mRNA vaccines, making it a valuable asset in global vaccination efforts, especially in regions with limited cold chain infrastructure. VACCINE IMMUNE RESPONSE ELICITATION For non-single-dose COVID-19 vaccines, the first and second doses play distinct and complementary roles in eliciting an effective immune response. The initial vaccine dose largely primes the immune system, providing the antigen in a way that stimulates initial antibody production and activates specific immune cells that lead to antigen-specific memory T and B cells. Because the vaccine is not an actual infection, it may not provide the needed cues to mount an optimal immune response. Thus, the second dose, or the booster, is crucial for amplifying and broadening this response. It significantly enhances the quantity and quality of neutralizing antibodies, solidifies memory B cell and T cell responses, and induces a more robust, durable immunity. The booster dose thus ensures a more sustained and effective immune response, including against virus variants (Chu et al., 2022). Table 2-1 presents a summary of antibody responses and T cell responses in humans for each U.S. COVID-19 vaccine. The immunogenicity of COVID-19 vaccines largely hinges on the adaptive immune system recognizing the specific spike protein fragments. B cell receptors (BCRs) on B cells and T cell receptors (TCRs) on T cells are key to this recognition when they interact with innate immune antigen-presenting cells. BCRs directly bind to epitopes on the spike protein, initiating B cell activation (Pettini et al., 2022). TCRs, however, recognize these epitopes when presented on Major Histocompatibility Complex (MHC) molecules by antigen-presenting cells (Yang et al., 2023). This dual recognition mechanism is essential for the coordinated activation of both humoral and cellular arms of the adaptive immune response (Teijaro and Farber, 2021) for viral proteins that are not superantigens. Following vaccination, B cell activation predominantly occurs in germinal centers (GCs) within secondary lymphoid organs (Figure 2-2), such as lymph nodes and the spleen. In general, antigen-activated B cells undergo somatic hypermutation, which introduces random mutations into their immunoglobulin genes (Laidlaw and Ellebedy, 2022; Turner et al., 2021) and leads to B cells with high-affinity antibodies for the spike protein. B cells with the highest affinity are selected and differentiated into long-lived plasma cells (LLPCs) and memory B cells (MBCs). LLPCs secrete neutralizing antibodies, some of which are capable of mediating sterilizing immunity, which prevents infection in the host including mucous membranes, and can potentially persist for years, continuously producing antibodies. MBCs quickly activate and give rise to a new wave of high-affinity antibody-secreting cells, providing rapid protection upon re- exposure to the virus (Sadarangani et al., 2021; Tam et al., 2016). For COVID vaccines, in time, mutations in the spike protein may result in lower affinity interaction between the antibodies induced by one strain and a mutated spike protein. The role of T cells, particularly CD4+ T cells, is multifaceted. T follicular helper (Tfh) cells, a subset of CD4+ T cells, are critical for the development of germinal center reactions and consequently for the maturation of B cell responses. Tfh cells assist B cells in the GCs by Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 36 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs providing necessary costimulatory signals and cytokines, facilitating the selection of high- affinity B cells. These interactions are crucial for developing both LLPCs and MBCs, and mRNA vaccines have been demonstrated to effectively induce Tfh cell responses, which are key to generating robust and long-lasting neutralizing immunity (Bettini and Locci, 2021; Pardi et al., 2018; Sadarangani et al., 2021). Clinically, however, immunity from COVID-19 vaccines is observed to wane over time (Menegale et al., 2023), necessitating booster doses to counteract this decline and to address the emergence of new, circulating common strains, thereby ensuring sustained protection against the virus. Cytotoxic CD8+ T cells, which directly eliminate virus-infected cells, are another crucial component. These cells are characterized by the release of cytotoxic molecules, such as granzyme B and perforin. Upon vaccination, polyfunctional antigen-specific CD8+ T cells increase; these produce inflammatory cytokines, which are critical signaling molecules in the immune system. These include IFNγ (interferon gamma), IL-2 (interleukin-2), and TNF (tumor necrosis factor). IFNγ plays a crucial role in activating and directing other immune cells, enhancing the overall immune response to the vaccine and the virus. IL-2 is vital for the growth, proliferation, and differentiation of T cells, ensuring a robust and sustained immune response. TNF is involved in systemic inflammation and capable of inducing apoptosis or cell death in virus-infected cells. These cells exhibit markers of cytotoxic activity and contribute to the overall defense against viral infection. The ability to activate CD8+ T cell responses varies among vaccine candidates, with some inducing strong responses in both small and large animal models, while others showing more variable results (Bettini and Locci, 2021; Creech et al., 2021). Immunological memory is a hallmark of the adaptive immune response and a key goal of vaccination. Most licensed vaccines, including those for COVID-19, confer protection by eliciting long-lasting antibody responses. The rapid and effective response to a pathogen upon re-exposure is primarily mediated by memory B and T cells. Memory B cells, upon re-exposure to the antigen, differentiate into antibody-secreting cells more quickly than naïve B cells, leading to a fast increase in antibody titers. Similarly, memory T cells, both CD4+ and CD8+, are primed to respond more rapidly and effectively than naïve T cells. Tfh cells are especially important in supporting memory B cell responses in GCs. They facilitate the selection of high-affinity memory B cells and their differentiation into LLPCs or MBCs (Pollard and Bijker, 2021). These interactions are critical for maintaining long-lasting immunity and providing rapid protection upon subsequent exposures to the virus. Upon activation in a future infection, memory B cells rapidly produce large amounts of antigen-specific antibody, which can neutralize viral infection/entry into host cells—reducing the severity of the infection. The duration of immunity conferred by COVID-19 vaccines and the potential need for booster doses are areas of ongoing research. Studies have shown that mRNA vaccines can induce robust CD8+ T cell responses characterized by key cytokines and cytotoxic markers upon rechallenge (Sadarangani et al., 2021; Teijaro and Farber, 2021). However, the longevity of these responses and persistence of memory T cells after vaccination is still under investigation. Some evidence suggests that the immune response elicited by these vaccines, particularly the generation of memory B and T cells, may be long lasting, but further studies are required to confirm the duration of this protection. Additionally, the need for booster doses may depend on factors such as the emergence of new viral variants/strains and the longevity of the vaccine- induced immune response (Teijaro and Farber, 2021). Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . IM M U N O L O G I C RE S P O N S E 37 PR E P U B L I C A T I O N C O P Y : Un c o r r e c t e d P r o o f s TA B L E 2 -1 Im m u n e R e s p o n s e s t o U . S . C O V I D - 19 V a c c i n e s Va c c i n e Pl a t f o r m Do s i n g Re g i m e n An t i b o d y R e s p o n s e s i n H u m a n s T C e l l R e s p o n s e s i n H u m a n s BN T 1 6 2 b 2 mR N A 30 μ g m R N A 2 d o s e s 21 d a y s a p a r t a S1 - bi n d i n g a n t i b o d y p r e s e n t a f t e r fi r s t d o s e , r e s p o n s e s i n c r e a s e d fo l l o w i n g t h e s e c o n d d o s e ; si g n i f i c a n t N a b w a s o n l y p r e s e n t af t e r s e c o n d d o s e b In c r e a s e s i n a n t i g e n - sp e c i f i c I F N γ + CD 4 + a n d C D 8 + T c e l l s a f t e r s e c o n d do s e ; p r e d o m i n a n c e o f I F N γ a n d I L -2 se c r e t i o n , c o m p a r e d w i t h I L - 4 , su g g e s t i n g T H 1 c e l l p o l a r i z a t i o n c mR N A -12 7 3 mR N A 10 0 μ g m R N A 2 d o s e s 28 d a y s a p a r t S- bi n d i n g a n t i b o d y d e t e c t e d 1 4 da y s a f t e r f i r s t d o s e , l e v e l s i n c r e a s e d sl i g h t l y b y 2 8 d a y s , w i t h ma r k e d in c r e a s e a f t e r s e c o n d d o s e d; m i n i m a l Na b p r e s e n t a f t e r f i r s t d o s e , p e a k a t 14 d a y s a f t e r s e c o n d d o s e e Si g n i f i c a n t i n c r e a s e s i n C D 4 + T c e l l s se c r e t i n g T H 1 t y p e c y t o k i n e s ( T N F > IL -2 > I F N γ ) a f t e r s e c o n d d o s e , s m a l l in c r e a s e s i n T N F - s e c r e t i n g a n d I L - 2 - se c r e t i n g c e l l s a f t e r f i r s t d o s e ; m i n i m a l ch a n g e i n T H 2 c e l l r e s p o n s e s ; l o w le v e l s o f C D 8 + r e s p o n s e s d Ad 2 6 . C O V 2 . S Vi r a l ve c t o r 5 × 1 0 10 vi r a l pa r t i c l e s 1 d o s e f S- bi n d i n g a n d n e u t r a l i z i n g a n t i b o d y pr e s e n t b y 2 8 d a y s a f t e r v a c c i n a t i o n in 9 9 % o f i n d i v i d u a l s a n d a n t i b o d y le v e l s s u s t a i n e d u n t i l a t l e a s t 8 4 d a y s po s t v a c c i n a t i o n g CD 4 + a n d C D 8 + T c e l l r e s p o n s e s pr e s e n t a t 1 4 - a n d 2 8 - da y s p o s t - va c c i n a t i o n , b a s e d o n p r e s e n c e o f CD 4 + a n d C D 8 + T c e l l s s e c r e t i n g I F N γ an d / o r I L - 2 a n d n o t I L - 4 o r I L -3 , su g g e s t i n g T H 1 c e l l p o l a r i z a t i o n o f t h e CD 4 + T c e l l r e s p o n s e g NV X -Co V 2 3 7 3 Pr o t e i n Su b u n i t 5 μ g pr o t e i n 2 d o s e s 21 d a y s a p a r t h S- bi n d i n g a n t i b o d y d e t e c t e d 2 1 da y s a f t e r f i r s t d o s e , w i t h a m a r k e d in c r e a s e a f t e r t h e s e c o n d d o s e ; s o m e Na b p r e s e n t a f t e r t h e f i r s t d o s e , w i t h a s i g n i f i c a n t i n c r e a s e b y 7 d a y s a f t e r se c o n d d o s e h CD 4 + T c e l l r e s p o n s e s p r e s e n t b y 7 da y s a f t e r s e c o n d d o s e , b a s e d o n I F N γ , IL -2 a n d T N F p r o d u c t i o n i n r e s p o n s e t o S p r o t e i n s t i m u l a t i o n , w i t h a s t r o n g b i a s to w a r d s a T H 1 c e l l p h e n o t y p e ; m i n i m a l TH 2 c e l l r e s p o n s e s ( a s m e a s u r e d b y I L - 5 a n d I L - 1 3 ) h Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 38 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y : Un c o r r e c t e d P r o o f s TA B L E 2 -1 Co n t i n u e d NO T E S : B N T 1 6 2 b 2 r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y P f i z e r -Bi o N T e c h u n d e r t h e n a m e C o m i r n a t y ® . m R N A -12 7 3 r e f e r s t o t h e CO V I D -19 v a c c i n e m a n u f a c t u r e d b y M o d e r n a u n d e r t h e n a m e S p i k e v a x ® . A d 2 6 . C O V 2 . S re f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y Ja n s s e n . N V X -Co V 2 3 7 3 r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y N o v a v a x . SO U R C E S : A d a p t e d f r o m S a d a r a n g a n i e t a l . , 2 0 2 1 . a Po l l a r d a n d B i j k e r , 2 0 2 1 . b Wa l s h e t a l . , 2 0 2 0 . c FD A , 2 0 2 1 a ; Sa d a r a n g a n i e t a l . , 2 0 2 1 . d FD A , 2 0 2 2 ; J a c k s o n e t a l . , 2 0 2 0 . e Wi d g e e t a l . , 2 0 2 1 . f Ma r f e e t a l . , 2 0 2 1 . g Sa d o f f e t a l . , 2 0 2 1 . h Ke e c h e t a l . , 2 0 2 0 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 39 PREPUBLICATION COPY: Uncorrected Proofs POSSIBLE MECHANISMS OF VACCINE-MEDIATED REACTIONS Although rare, vaccine-mediated harms can range from mild, transient reactions to more serious conditions, underscoring the importance of ongoing safety monitoring and research. Certain of the most common vaccine-associated harms can arise from a few different immunological mechanisms, some of which are briefly discussed next (Table 2-2). Immediate-type hypersensitivity reactions are rapid immunological responses observed in certain individuals following vaccination. Mast cells and basophils play a crucial role; they become activated by IgE when individuals are re-exposed to the same antigen during vaccination (Stone et al., 2019), which triggers degranulation and the release of various mediators, such as histamine, leukotrienes, prostaglandins, and cytokines, including IL-4 and IL-5. The clinical manifestation ranges from urticaria (hives) to the more severe and potentially life-threatening anaphylaxis (McLeod et al., 2015). In contrast, delayed-type hypersensitivity reactions involve a different immune pathway. T cells, particularly CD4+ helper T cells, are central to these reactions. Upon exposure to an antigen that the immune system has seen before, T cells secrete cytokines, such as Interferon- gamma (IFNγ), IL-2, and tumor necrosis factor-alpha (TNF-α). The symptoms associated with this reaction, such as rash, fever, and joint pain, typically develop days to weeks after vaccination, distinguishing them from the immediate-type reactions (Biedermann et al., 2000). Autoimmune reactions in the context of vaccination encompass a variety of plausible mechanisms (Chen et al., 2022a; Lamprinou et al., 2023): ● Molecular mimicry, where vaccine antigens closely resemble the body’s own proteins, potentially leading to the production of autoantibodies or autoreactive T cells that target self-tissues (Segal and Shoenfeld, 2018). ● Bystander activation, when localized inflammation exposes self-antigens, leading to the activation of previously dormant self-reactive lymphocytes. ● Epitope spreading, particularly with repeat vaccinations, where the initial immune response to vaccine antigens broadens to include self-antigens. ● Polyclonal activation and adjuvant-induced autoimmunity, where intense immune stimulation, potentially exacerbated by adjuvants, overcomes the tolerance to self- antigens, resulting in autoimmunity. A current and significant concern is vaccine-induced immune thrombotic thrombocytopenia (VITT), an extremely rare condition characterized by forming antibodies against platelet factor 4 (PF4). This activates platelets and immune cells producing anti-PF4 antibodies (Dabbiru et al., 2023). The role of complement activation in promoting a prothrombotic state is also being explored (see Chapter 5). Vaccine-Associated Enhanced Disease (VAED) and Antibody-Dependent Enhancement (ADE) are critical considerations in vaccine development, particularly highlighted by historical challenges with the formalin-inactivated Respiratory Syncytial Virus (RSV) vaccine (Acosta et al., 2015). VAED encompasses a spectrum of phenomena where vaccination paradoxically exacerbates the disease upon exposure to the natural pathogen, mediated through mechanisms such as ADE. In ADE, non-neutralizing or suboptimal antibodies generated by the vaccine facilitate the pathogen's entry into host cells via Fc receptors, leading to increased viral replication and severe disease manifestations (Gartlan et al., 2022). The formalin-inactivated Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 40 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs RSV vaccine is a notable example where immunization induced antibodies that not only failed to confer protection but also potentiated respiratory disease upon subsequent natural RSV infection. This outcome was partly attributed to the vaccine eliciting a skewed Th2-type immune response, promoting eosinophilic infiltration and severe lung pathology, rather than a protective Th1-type response (Gartlan et al., 2022). Additionally, immune complexes formed by the vaccine-induced antibodies could activate complement pathways, contributing to tissue damage. Furthermore, general vaccine reactions encompass a wide array of immune responses. These involve the activation of antigen-presenting cells (APCs), B cells, and T cells. Cytokines, such as IL-1, IL-6, IL-12, and TNF-α, play a significant role in the initial immune response to vaccines, contributing to both their protective effects and potential harms. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . IM M U N O L O G I C RE S P O N S E 41 PR E P U B L I C A T I O N C O P Y : Un c o r r e c t e d P r o o f s TA B L E 2 -2 Va c c i n e -Me d i a t e d R e a c t i o n s a n d T h e i r M e c h a n i s m s Ty p e o f R e a c t i o n Im m u n e C e l l s In v o l v e d Pl a u s i b l e M e c h a n i s m s Cl i n i c a l Ma n i f e s t a t i o n Ti m e o f O n s e t Im m e d i a t e hy p e r s e n s i t i v i t y Ma s t c e l l s , ba s o p h i l s Ig E -me d i a t e d m a s t c e l l / b a s o p h i l ac t i v a t i o n i n d u c e d b y p r e v i o u s ex p o s u r e t o a n t i g e n s i n t h e v a c c i n e , le a d i n g t o d e g r a n u l a t i o n a n d r e l e a s e of h i s t a m i n e , l e u k o t r i e n e s , pr o s t a g l a n d i n s , c y t o k i n e s (I L -4, I L -5) Ur t i c a r i a ( h i v e s ) to a n a p h y l a x i s Ra p i d , p o s t v a c c i n a t i o n De l a y e d hy p e r s e n s i t i v i t y CD 4 + h e l p e r T ce l l s Se c r e t i o n o f c y t o k i n e s ( I F N -γ, I L -2, TN F -α) u p o n a c t i v a t i o n Ra s h , f e v e r , jo i n t p a i n Da y s t o w e e k s Au t o i m m u n e re a c t i o n s Va r i o u s Mo l e c u l a r m i m i c r y , b y s t a n d e r ac t i v a t i o n , e p i t o p e s p r e a d i n g , po l y c l o n a l a c t i v a t i o n , a d j u v a n t - in d u c e d a u t o i m m u n i t y , a n d o t h e r s Va r i e s Va r i e s Va c c i n e -in d u c e d im m u n e t h r o m b o t i c th r o m b o c y t o p e n i a (V I T T ) Pl a t e l e t s , im m u n e c e l l s pr o d u c i n g a n t i - PF 4 a n t i b o d i e s Fo r m a t i o n o f a n t i b o d i e s a g a i n s t pl a t e l e t f a c t o r 4 ( P F 4 ) , co m p l e m e n t ac t i v a t i o n Th r o m b o s i s , th r o m b o c y t o p e n i a Po s t v a c c i n a t i o n ( v a r i a b l e ) Va c c i n e -As s o c i a t e d En h a n c e d D i s e a s e (V A E D ) B c e l l s , T h 2 sk e w e d im m u n i t y No n -ne u t r a l i z i n g o r s u b o p t i m a l an t i b o d i e s , g e n e r a t e d i n r e s p o n s e t o a va c c i n e , f a c i l i t a t e v i r a l en t r y i n t o h o s t ce l l s t h r o u g h F c r e c e p t o r s o r co m p l e m e n t r e c e p t o r s . Wo r s e n i n g o f di s e a s e s / sy m p t o m s Up o n e x p o s u r e t o n a t u r a l vi r u s a f t e r v a c c i n a t i o n Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 42 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y : Un c o r r e c t e d P r o o f s TA B L E 2 -2 Co n t i n u e d Ty p e o f R e a c t i o n Im m u n e C e l l s In v o l v e d Pl a u s i b l e Me c h a n i s m s Cl i n i c a l Ma n i f e s t a t i o n Ti m e o f O n s e t Ot h e r AP C s , B c e l l s , T c e l l s Ac t i v a t i o n o f i m m u n e c e l l s a n d re l e a s e o f c y t o k i n e s ( I L -1 , I L - 6 , I L - 12 , T N F - α) Va r i e s Po s t v a c c i n a t i o n SO U R C E S : C h e n e t a l . , 2 0 2 2 b ; D a b b i r u e t a l . , 2 0 2 3 ; L a m p r i n o u e t a l . , 2 0 2 3 ; S e g a l a n d S h o e n f e l d , 2 0 1 8 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 43 PREPUBLICATION COPY: Uncorrected Proofs ADJUVANTS Adjuvants in vaccines serve to enhance the body’s immune response to an antigen, ensuring a stronger and longer-lasting immunity by activating TLRs on antigen-presenting cells to stimulate a strong innate immune response that produces a strong adaptive immune response. For example, aluminum salts create a depot effect for sustained antigen release, and oil-in-water emulsions, such as MF59, increase cytokine release and antigen uptake (Wilkins et al., 2017). Adjuvants such as AS01, AS02, AS03, and saponins stimulate APCs, such as dendritic cells, to activate T cells, and CpG oligodeoxynucleotides activate Toll-like receptor 9 (TLR9) (Facciola et al., 2022). These mechanisms, although crucial for vaccine efficacy, can sometimes lead to adverse reactions, primarily localized ones, such as inflammation and soreness, due to heightened immune activation at the site of injection. Table 2-3 lists some of the most commonly used adjuvants and their mechanisms of action. Matrix-M® is a saponin-based adjuvant in NVX-CoV2373, which is the only specifically adjuvanted COVID-19 vaccine. It consists of Quillaja Saponaria Molina extracts, known for their ability to stimulate both the innate and adaptive arms of the immune system. It enhances immune responses by activating antigen-presenting cells and boosting cytokine production, which facilitates a stronger T cell and antibody response to the vaccine antigen (Stertman et al., 2023). Its mechanism of action increases the vaccine’s efficacy, but like other adjuvants, it can also contribute to or cause reactions. In the United States, FDA approves adjuvants only as components of vaccines, not as stand-alone products, because their properties can vary based on their concentration and interaction with other ingredients in the vaccine formulation. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 44 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y : Un c o r r e c t e d P r o o f s TA B L E 2 -3 Mo s t C o m m o n l y U s e d A d j u v a n t s i n V a c c i n e s Ad j u v a n t Me c h a n i s m o f A c t i o n Al u m i n u m s a l t s ( a l u m ) Cr e a t e s a d e p o t e f f e c t , s l o w l y r e l e a s i n g a n t i g e n a n d e n h a n c i n g a n t i g e n u p t a k e b y an t i g e n -pr e s e n t i n g c e l l s . MF 5 9 (o i l -in -wa t e r e m u l s i o n ) In c r e a s e s c y t o k i n e r e l e a s e a n d a n t i g e n u p t a k e , s t i m u l a t i n g a s t r o n g e r i m m u n e re s p o n s e . AS 0 4 ( a l u m i n u m s a l t + m o n o p h o s p h o r y l l i p i d (M P L ) A ) Co m b i n e s a l u m ’ s d e p o t e f f e c t w i t h M P L t o e n h a n c e t h e i m m u n e r e s p o n s e . M P L ac t i v a t e s TL R 4 . Cp G o l i g o d e o x y n u c l e o t i d e s Ac t i v a t e s T L R 9 , e n h a n c i n g t h e i m m u n e r e s p o n s e t o s p e c i f i c p a t h o g e n s . AS 0 1 ( l i p o s o m e b a s e d ) St i m u l a t e s d e n d r i t i c c e l l s a n d T c e l l r e s p o n s e s , e n h a n c i n g b o t h i n n a t e a n d ad a p t i v e i m m u n i t y . Vi r o s o m e s Mi m i c s v i r a l in f e c t i o n , e n h a n c i n g t h e i m m u n e s y s t e m ’ s r e c o g n i t i o n a n d r e s p o n s e to t h e a n t i g e n . QS -21 ( s a p o n i n b a s e d ) En h a n c e s a n t i g e n p r e s e n t a t i o n a n d s t i m u l a t e s b o t h h u m o r a l a n d c e l l u l a r i m m u n e re s p o n s e s . Li p o p o l y s a c c h a r i d e ( L P S ) Ac t i v a t e s T L R 4 , w h i c h l e a d s t o st r o n g a n t i b o d y r e s p o n s e s b y a c t i v a t i n g T h 2 c e l l s . Po l y -IC L C ( p o l y i n o s i n i c -po l y c y t i d y l i c a c i d ) Mi m i c s v i r a l R N A , s t i m u l a t i n g a s t r o n g i m m u n e r e s p o n s e . Ma t r i x -M® ( s a p o n i n b a s e d ) Ac t i v a t e s a n t i g e n -pr e s e n t i n g c e l l s a n d b o o s t s c y t o k i n e p r o d u c t i o n , en h a n c i n g T ce l l a n d a n t i b o d y r e s p o n s e s . Ad j u v a n t s y s t e m 0 3 ( A S 0 3 , o i l -in -wa t e r em u l s i o n ) Co n t a i n s s q u a l e n e , D L -α-to c o p h e r o l , a n d p o l y s o r b a t e 8 0 , e n h a n c i n g i m m u n e re s p o n s e v i a c y t o k i n e m o d u l a t i o n . SO U R C E S : S t e r t m a n e t a l . , 2 0 2 3 ; W i l k i n s e t a l . , 2 0 1 7 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 45 PREPUBLICATION COPY: Uncorrected Proofs Potential harms of vaccination necessitate a thorough investigation of mechanisms. Examining their immune response will help investigators gain insights into possible mechanisms of vaccine-related harms. Through an examination of clinical trials, epidemiology studies, case reports, preclinical in vivo and in silico work, and insights from animal models, the committee has delved into possible mechanisms that may contribute to adverse events. Understanding these mechanisms is paramount in ensuring the safety and well-being of individuals receiving COVID-19 vaccines. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. 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Native-like SARS-CoV-2 spike glycoprotein expressed by ChAdOx1 nCoV-19/AZD1222 vaccine. ACS Central Science 7(4):594–602. https://doi.org/10.1021/acscentsci.1c00080. Welsh, J. 2021. Coronavirus variants—will new mRNA vaccines meet the challenge? Engineering 7(6):712–714. https://doi.org/10.1016/j.eng.2021.04.005. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. IMMUNOLOGIC RESPONSE 51 PREPUBLICATION COPY: Uncorrected Proofs Widge, A. T., N. G. Rouphael, L. A. Jackson, E. J. Anderson, P. C. Roberts, M. Makhene, J. D. Chappell, M. R. Denison, L. J. Stevens, A. J. Pruijssers, A. B. McDermott, B. Flach, B. C. Lin, N. A. Doria- Rose, S. O’Dell, S. D. Schmidt, K. M. Neuzil, H. Bennett, B. Leav, M. Makowski, J. Albert, K. Cross, V. V. Edara, K. Floyd, M. S. Suthar, W. Buchanan, C. J. Luke, J. E. Ledgerwood, J. R. Mascola, B. S. Graham, J. H. Beigel, and mRNA-1273 Study Group 2021. Durability of responses after SARS-CoV-2 mRNA-1273 vaccination. New England Journal of Medicine 384(1):80–82. https://doi.org/10.1056/NEJMc2032195. Wilkins, A. L., D. Kazmin, G. Napolitani, E. A. Clutterbuck, B. Pulendran, C. A. Siegrist, and A. J. Pollard. 2017. AS03- and Mf59-adjuvanted influenza vaccines in children. Frontiers in Immunology 8:1760. https://doi.org/10.3389/fimmu.2017.01760. Wrapp, D., N. Wang, K. S. Corbett, J. A. Goldsmith, C. L. Hsieh, O. Abiona, B. S. Graham, and J. S. McLellan. 2020. Cryo-em structure of the 2019-nCoV spike in the prefusion conformation. bioRxiv. https://doi.org/10.1101/2020.02.11.944462. Xiaojie, S., L. Yu, Y. Lei, Y. Guang, and Q. Min. 2020. Neutralizing antibodies targeting SARS-CoV-2 spike protein. Stem Cell Research 50:102125. https://doi.org/10.1016/j.scr.2020.102125. Yang, G., J. Wang, P. Sun, J. Qin, X. Yang, D. Chen, Y. Zhang, N. Zhong, and Z. Wang. 2023. SARS- CoV-2 epitope-specific T cells: Immunity response feature, TCR repertoire characteristics and cross-reactivity. Frontiers in Immunology 14:1146196. https://doi.org/10.3389/fimmu.2023.1146196. Zost, S. J., P. Gilchuk, R. E. Chen, J. B. Case, J. X. Reidy, A. Trivette, R. S. Nargi, R. E. Sutton, N. Suryadevara, E. C. Chen, E. Binshtein, S. Shrihari, M. Ostrowski, H. Y. Chu, J. E. Didier, K. W. MacRenaris, T. Jones, S. Day, L. Myers, F. Eun-Hyung Lee, D. C. Nguyen, I. Sanz, D. R. Martinez, P. W. Rothlauf, L. M. Bloyet, S. P. J. Whelan, R. S. Baric, L. B. Thackray, M. S. Diamond, R. H. Carnahan, and J. E. Crowe, Jr. 2020. Rapid isolation and profiling of a diverse panel of human monoclonal antibodies targeting the SARS-CoV-2 spike protein. Nature Medicine 26(9):1422–1427. https://doi.org/10.1038/s41591-020-0998-x. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 52 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY: Uncorrected Proofs Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. PREPUBLICATION COPY—Uncorrected Proofs 3 Neurologic Conditions and COVID-19 Vaccines This chapter describes the potential relationship between COVID-19 vaccines and potential neurological harms Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyneuropathy, Bell’s palsy (BP), transverse myelitis (TM), chronic headache, and postural orthostatic tachycardia syndrome (POTS) (see Boxes 3-1 through 3-6 for all conclusions in this chapter). GUILLAIN-BARRÉ SYNDROME BOX 3-1 Conclusions for Guillain-Barré syndrome Conclusion 3-1: The evidence favors rejection of a causal relationship between the BNT162b2 vaccine and Guillain-Barré syndrome. Conclusion 3-2: The evidence favors rejection of a causal relationship between the mRNA- 1273 vaccine and Guillain-Barré syndrome. Conclusion 3-3: The evidence favors acceptance of a causal relationship between the Ad26.COV2.S vaccine and Guillain-Barré syndrome. Conclusion 3-4: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and Guillain-Barré syndrome. Background GBS is an acute, monophasic, immune-mediated disorder, or group of disorders, that primarily affects the peripheral nerves and roots. The typical clinical features include progressive symmetric muscle weakness and absent or depressed deep tendon reflexes. Patients may also experience tingling or prickling sensations (paresthesia) along with autonomic dysfunction, including fluctuations in blood pressure, heart rate, and respiratory distress. Cranial nerve involvement can result in facial weakness, difficulty swallowing, and speech problems, and some individuals experience significant pain, particularly in the back or legs. Symptoms usually progress over 1–2 weeks and generally plateau before 4 weeks (Fokke et al., 2014). Diagnosing GBS is a multifaceted process that involves a comprehensive clinical evaluation, cerebrospinal fluid (CSF) analysis, and electrodiagnostic studies. A thorough clinical history and neurological examination are critical to assess the pattern of weakness and reflex Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 54 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs abnormalities. Analysis of CSF often reveals elevated protein levels without a significant increase in white blood cells. Electrophysiological tests can confirm the diagnosis by revealing evidence of nerve demyelination in demyelinating variants of GBS and identifying pathological changes affecting both the roots and nerves. GBS is a relatively rare disease, with a global incidence of 0.81–1.91 cases per 100,000 person-years (Shahrizaila et al., 2021). The U.S. incidence of GBS is generally in line with the global average, with an estimated 1–2 cases per 100,000 individuals each year (Bragazzi et al., 2021). Although all age groups are affected, the incidence increases by approximately 20 percent with every 10-year increase beyond the first decade of life, with a peak incidence reported between 50–69 years and a slight male predominance (Leonhard et al., 2022). The pathophysiology of GBS remains incompletely understood and is likely heterogeneous, reflecting phenotypic variability among what is likely a group of related disorders rather than a single nosological entity. Despite this heterogeneity, more than two-thirds of patients report a history of upper respiratory tract or gastrointestinal infection weeks before the onset of neurologic symptoms, suggesting infection plays an important pathogenic role in all GBS variants (Leonhard et al., 2022). Although GBS is a global disease, regional differences occur in the distribution of variants. Demyelinating forms dominate in Europe and North America, but acute inflammatory demyelinating polyradiculoneuropathy (AIDP) accounts for 80–90 percent of cases and is characterized by ascending limb weakness. Other demyelinating variants with prominent and early cranial nerve involvement affecting eye movements and facial muscles, including the Miller-Fisher and facial diplegia with limb paresthesia variant, are rare. Axonal subtypes, such as acute motor axonal neuropathy (AMAN), dominate in Asia, particularly Bangladesh and north China (Leonhard et al., 2022). Seasonal variation of incidence track with infections. The risk is higher during the winter, particularly in Europe and North America, where it is associated primarily with upper respiratory infections. A summer peak occurs in Northern China, India, Bangladesh, and Latin America, where diarrheal illnesses can be more common. Incidence can also rise during outbreaks of infection, such as with Zika virus in South America or other arthropod infections, such as dengue and chikungunya (Shahrizaila et al., 2021). Globally, commonly implicated pathogens include Campylobacter jejuni, cytomegalovirus, Epstein-Barr virus, Mycoplasma pneumoniae, Haemophilus influenzae, influenza A virus, and Zika virus (Shahrizaila et al., 2021). C. Jejuni is the most commonly and extensively reported, and robust evidence suggests that molecular mimicry between microbial antigens and nerves is implicated in developing GBS. In addition to infection, GBS cases after vaccination have also been reported, especially with the 1976 swine-influenza and seasonal 2009 H1N1 monovalent influenza vaccines. However, the overall risk of influenza vaccines if present at all appears to be small, approximately 1–2 excess cases of GBS per million people vaccinated (Vellozzi et al., 2014). While some have reported an increased risk of GBS after SARS-CoV-2 infection, the actual incidence of GBS decreased during the pandemic, possibly due to an overall reduction in other communicable diseases (Keddie et al., 2021). The latency period between exposure to a triggering event (infection or vaccination) and GBS can vary, but it typically occurs within a few days to a few weeks. It is crucial to understand that not everyone exposed to these risk factors will develop GBS, and the exact mechanisms continue to be the subject of ongoing research. The epidemiology of GBS can be influenced by various factors, including changes in diagnostic techniques, vaccination practices, and evolving patterns of infectious diseases, so ongoing surveillance and research are crucial to continually monitor and understand it. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 55 PREPUBLICATION COPY—Uncorrected Proofs Mechanisms GBS is heterogeneous because it is likely a group of related disorders. Demyelinating variants, such AIDP, differ from axonal variants, such as AMAN, in both the range and extent of pathological changes. Nevertheless, nerve injury appears to be immune mediated, with antecedent infection being a common potential trigger. Autopsy studies demonstrate infiltrates of lymphocytes and macrophages involved in macrophage-mediated demyelination (Asbury et al., 1969; Wanschitz et al., 2003). Complement deposition can be demonstrated within the endoneurium, on the surface of myelinated fibers, and on mononuclear cells at sites of myelin breakdown, particularly in acute cases of less than 4 weeks duration, suggesting a role for antibody-mediated injury, whereas granzyme-expressing CD8+ T cells (i.e., cytotoxic T cells) are described in cases of longer duration (Wanschitz et al., 2003). By contrast, patients with AMAN demonstrate primary axonal injury with a paucity of inflammatory infiltrates or demyelination. IgG and complement-mediated humoral immune response are directed against epitopes in the axonal membrane. Animal models of GBS have been generated by immunizing rats with myelin proteins, galactocerebroside, adoptive transfer of myelin-specific T cells (AIDP), or immunization with GM1 ganglioside, resulting in circulating anti-GM1 antibodies (AMAM) (Figure 3-1) (Shahrizaila et al., 2021). These animal models implicate T cells and macrophages in AIDP but suggest that autoantibodies may play a greater role in AMAM (Shahrizaila et al., 2021). The mechanism of antibody-mediated damage may include interference with ion channel function, complement-dependent cytotoxicity, and/or interference with nerve regeneration; different clinical subtypes of GBS are associated with different anti-ganglioside antibodies (Shahrizaila et al., 2021). FIGURE 3-1 Overview of the pathogenesis and therapeutic targets of the two major Guillain-Barré syndrome subtypes. SOURCE: Shahrizaila et al., 2021. Evidence for molecular mimicry is best supported for C. jejuni-associated AMAN, where the reasoning is as follows (Yuki et al., 2004): Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 56 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs ● Patients with GBS after C. jejuni, but not patients with C. jejuni enteritis, have antibodies to GM1 ganglioside in their serum (Sheikh et al., 1998). ● The specific serotype of C. jejuni most commonly isolated from patients with GBS (PEN19) is rare in patients with C. jejuni enteritis. ● The GM1 ganglioside has an antigenic similarity with the lipopolysaccharide of C. jejuni serotype PEN19 (Yuki et al., 1993). ● Rabbits sensitized to C. jejuni LPS develop AMAM and flaccid limb weakness with pathological findings similar to GBS. ● Anti-GM1 IgG from patients with GBS can block muscle action potentials in muscle- spinal cord coculture, although they do not induce weakness when injected into mice (Yuki et al., 2004). C. jejuni infection can also generate antibodies against GQ1b gangliosides, which are associated with the Miller-Fisher GBS variant (Jacobs et al., 1997). Anti-ganglioside antibodies, however, are not found in association with all GBS variants. In addition, as mentioned in the background section, GBS is associated with a variety of pathogens, including potentially SARS- CoV-2, arguing against molecular mimicry as the single unifying mechanism in all forms of it. A few in silico studies have sought peptide antigens in SARS-CoV-2 with the potential to induce antibodies that cross-react with proteins in the peripheral or central nervous system, thereby activating complement and mediating neuronal damage (Chen et al., 2022b; Kadkhoda, 2022). One such study demonstrated similarity between a peptide in SARS-CoV2 and the NCAM L1–like protein in the myelin sheath and argued that cross-reactive antibodies might explain GBS after infection (Kadkhoda, 2022; Morsy, 2020). However, the shared peptide was in the SARS-CoV-2 envelope protein, not the spike protein, and would not provide mechanistic evidence for GBS occurring after COVID-19 vaccination. Epidemiological evidence suggests a possible association between adenoviral vector (AV) COVID-19 vaccines and GBS but not for the messenger ribonucleic acid (mRNA) vaccines (Hanson et al., 2022; Keh et al., 2023). This suggests the possibility of a platform-specific mechanism or immune response as opposed to one related to immune responses to the spike protein itself (such as molecular mimicry) (Rzymski, 2023). One study found high levels of complement-fixing antibodies to cytomegalovirus in a cohort of patients with GBS but no comparable antibodies to adenovirus in the same patients (Dowling et al., 1977), and adenovirus has not been historically linked with GBS in epidemiological studies. This suggests that natural adenoviral infection may not be associated with GBS. ChAdOx1-S 1 has high affinity for the coxsackie and adenoviral receptor (CAR), whereas HAdV26 has much lower CAR affinity (Baker et al., 2021; Hemsath et al., 2022; Rzymski, 2023). CAR is widely expressed in the body, including the central nervous system (Zussy et al., 2016); however, whether it is expressed in the peripheral nervous system has not been established. Therefore, it is unknown whether ChAdOx1-S could target peripheral nerves directly. Epidemiological Evidence Clinical trial results submitted to FDA for Emergency Use Authorization and full approval do not indicate a signal regarding GBS and any of the vaccines under study (FDA, 2021, 2023a, 2023b, 2023c). Table 3-1 presents eight studies that contributed to the causality assessment. 1 Refers to the COVID-19 vaccine manufactured by Oxford-AstraZeneca. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . NE U R O L O G I C CO N D I T I O N S 57 PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -1 Ep i d e m i o l o g i c a l S tu d i e s i n t h e Gu i l l a i n -Ba r r é S y n d r o m e Ev i d e n c e R e v i e w Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) Ha n s o n e t al . (2 0 2 2 ) Co h o r t / va c c i n a t e d co n c u r r e n t co m p a r a t o r s an d hi s t o r i c a l co n t r o l s US VS D / EM R (p h y s i c i a n ad j u d i c a t e d ) BN T 1 6 2 b 2 ≥1 2 ye a r s 8. 8 mi l l i o n do s e s 9 c a s e s me e t i n g Br i g h t o n cr i t e r i a 1 –3 du r i n g 1 –21 - da y r i s k p e r i o d RR 2 0 . 5 6 (6 . 9 4 –64 . 6 6 ) 15 . 5 e x c e s s ca s e s i n r i s k in t e r v a l p e r mi l l i o n d o s e s o f Ad 2 6 . C O V 2 . S co m p a r e d t o m R N A va c c i n e s mR N A -12 7 3 5. 8 mi l l i o n do s e s 9 c a s e s me e t i n g Br i g h t o n cr i t e r i a 1 –3 du r i n g 1 –21 - da y r i s k p e r i o d Ad 2 6 . C O V 2 .S 48 3 , 0 5 3 do s e s 8 c a s e s me e t i n g Br i g h t o n cr i t e r i a 1 –3 du r i n g 1 –21 - da y r i s k p e r i o d Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 58 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -1 Co n t i n u e d Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s ( 9 5 % CI ) Ke h e t a l . (2 0 2 3 ) Co h o r t / va c c i n a t e d GB S c a s e s UK Na t i o n a l im m u n i z a t i o n da t a b a s e / Na t i o n a l im m u n e - gl o b u l i n da t a b a s e BN T 1 6 2 b 2 ≥1 8 ye a r s 11 . 5 mil l i o n do s e s 21 c a s e s in 0 –42 d a y s No e x c e s s ri s k o f G B S ob s e r v e d 0 –42 da y s f o l l o w i n g BN T 1 6 2 b 2 va c c i n e co m p a r e d to v a c c i n a t e d ca s e s i n co n t r o l p e r i o d mR N A -12 7 3 30 0 , 0 0 0 do s e s 1 Ch A d O x 1 -S 20 . 3 mi l l i o n do s e s 17 6 Kl e i n e t a l . (2 0 2 1 ) Co h o r t / va c c i n a t e d co n c u r r e n t co m p a r a t o r s US VS D / EM R BN T 1 6 2 b 2 ≥1 6 ye a r s 6. 8 mi l l i o n dos e s BN T 1 2 b 2 a n d mR N A -12 7 3 co m b i n e d : 10 BN T 1 2 b 2 a n d mR N A -12 7 3 co m b i n e d RR 0 . 7 0 (0 . 2 2 –2. 3 1 ) mR N A -12 7 3 5. 1 mi l l i o n do s e s Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . NE U R O L O G I C CO N D I T I O N S 59 PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) Li e t a l . (2 0 2 2 ) Co h o r t / hi s t o r i c a l ba c k g r o u n d UK a n d Sp a i n Pr i m a r y c a r e da t a b a s e s li n k e d t o ho s p i t a l d a t a / EM R BN T 1 6 2 b 2 (U K a n d Sp a i n ) ≥1 8 ye a r s UK Do s e 1 : 1. 7 m i l l i o n va c c i n e e s Do s e 2 : 1. 2 m i l l i o n va c c i n e e s Sp a i n Do s e 1 : 1. 9 m i l l i o n va c c i n e e s Do s e 2 : 1. 3 m i l l i o n va c c i n e e s UK Do s e 1 : < 5 Do s e 2 : < 5 Sp a i n Do s e 1 : 5 Do s e 2 : < 5 UK N/ A Sp a i n Do s e 1 : SI R 0 . 7 9 (0 . 3 3 –1. 9 1 ) mR N A -12 7 3 (S p a i n o n l y ) Do s e 1 : 24 4 , 9 1 3 va c c i n e e s Do s e 2 : 16 0 , 2 1 3 va c c i n e e s Do s e 1 : 0 Do s e 2 : < 5 ca s e s N/ A Ad 2 6 . C O V 2 .S ( S p a i n on l y ) Do s e 1 : 12 0 , 7 3 1 va c c i n e e s Do s e 1: 0 N/ A Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 60 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s Ch A d O x 1 -S (U K a n d Sp a i n ) UK Do s e 1 : 3. 8 m i l l i o n va c c i n e e s Do s e 2 : 1. 1 m i l l i o n va c c i n e e s Sp a i n Do s e 1 : 59 2 , 8 6 0 va c c i n e e s Do s e 2 : 1. 3 m i l l i o n va c c i n e e s UK Do s e 1 : 11 Do s e 2 : < 5 Sp a i n Do s e 1 : < 5 Do s e 2 : 0 UK SI R 0 . 7 4 (0 . 4 1 –1. 3 3 ) Sp a i n N/ A Lo o e t a l . (2 0 2 1 ) Ca s e -co n t r o l st u d y / hi s t o r i c a l ba c k g r o u n d UK EM R (p h y s i c i a n ad j u d i c a t e d ) BN T 1 6 2 b 2 ≥1 6 ye a r s 24 p a t i e n t s ad m i t t e d wi t h a c u t e on s e t po l y r a d i c u l on e u r o p a t h y b e t w e e n Ja n u a r y – Ju n e 2 0 2 1 1 2. 6 -fo l d (1 . 9 8 –3. 5 1 ) in c r e a s e i n ad m i s s i o n s co m p a r e d wi t h t h e av e r a g e f o r th e s a m e p e r i o d in t h e p r e v i o u s 3 y e a r s mR N A - 12 7 3 1 Ch A d O x 1 - S 14 Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . NE U R O L O G I C CO N D I T I O N S 61 PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -1 Co n t i n u e d Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) Mo r c i a n o e t al . ( 2 0 2 2 ) Co h o r t / s e l f - co n t r o l l e d It a l y Mu l t i -re g i o n a l da t a b a s e s / E M R BN T 1 6 2 b 2 ≥1 2 ye a r s 10 . 8 mi l l i o n va c c i n e e s Do s e 1 : 1 9 Do s e 2 : 3 0 Do s e 1 : RI 0 . 8 5 (0 . 4 9 –1. 4 8 ) Do s e 2 : RI 1. 3 0 (0 . 8 0 –2. 1 0 ) mR N A - 12 7 3 1. 7 mi l l i o n va c c i n e e s Do s e 1 : 7 Do s e 2 : 5 Do s e 1 : RI 6. 8 3 (2 . 1 4 –21 . 8 5 ) Do s e 2 : RI 7 . 4 1 (2 . 3 5 –23 . 3 8 ) Ad 2 6 . C O V 2. S 58 1 , 7 9 6 va c c i n e e s Do s e 1 : 7 Do s e 1 : RI 1 . 9 4 (0 . 3 2 –11 . 6 9 ) Ch A d O x 1 - S 2. 9 mi l l i o n va c c i n e e s Do s e 1 : 3 4 Do s e 2 : 6 Do s e 1 : RI 6 . 5 2 (2 . 8 8 –14 . 7 7 ) Do s e 2 : RI 3. 5 6 (0 . 3 1 –40 . 2 9 ) Pa t o n e e t a l . (2 0 2 1 ) Co h o r t / s e l f - co n t r o l l e d En g l a n d En g l i s h im m u n i z a t i o n re c o r d s /E M R BN T 1 6 2 b 2 ≥1 6 ye a r s 12 . 1 mi l l i o n va c c i n e e s 34 c a s e s du r i n g 1 –28 da y s r i s k in t e r v a l IR R 0 . 8 6 (0 . 5 4 –1. 3 6 ) Ch A d O x 1 - S 20 . 4 mi l l i o n va c c i n e e s 15 3 1–28 d a y s IR R 2 . 0 4 (1 . 6 0 –2. 6 0 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 62 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -1 Co n t i n u e d Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) St u r k e n b o o m et a l . ( 2 0 2 2 ) Co h o r t / b a c k gr o u n d r a t e Eu r o p e a n co u n t r i e s Pr i m a r y a n d se c o n d a r y c a r e da t a b a s e s / E M R BN T 1 6 2 b 2 Va r i e d 6. 5 mi l l i o n do s e s 16 IR R 1 . 1 0 (0 . 5 6 –2. 1 5 ) mR N A - 12 7 3 72 7 , 0 4 7 do s e s No c a s e s N/ A Ad 2 6 . C O V 2. S 24 2 , 3 4 9 do s e s 2 IR R 5 . 6 5 (1 . 4 –22 . 8 3 ) Ch A d O x 1 - S 4. 6 mi l l i o n do s e s 15 IR R 1 . 4 3 (0 . 8 5 –2. 4 0 ) Wa l k e r e t a l . (2 0 2 2 ) Se l f - co n t r o l l e d co h o r t UK EM R BN T 1 6 2 b 2 ≥1 8 ye a r s 5. 7 mi l l i o n va c c i n e e s 28 3 IR R 1 . 0 0 (0 . 6 1 –1. 6 4 ) mR N A - 12 7 3 25 5 , 4 4 6 va c c i n e e s No c a s e s N/ A Ch A d O x 1 - S 7. 8 mi l l i o n va c c i n e e s 51 7 IR R 2 . 8 5 (2 . 3 3 –3. 4 7 ) NO T E S : B N T 1 6 2 b 2 r e f e r s t o t h e CO V I D -19 v a c c i n e m a n u f a c t u r e d b y P f i z e r -Bi o N T e c h u n d e r t h e n a m e C o m i r n a t y ® . m R N A -12 7 3 r e f e r s t o t h e CO V I D -19 v a c c i n e m a n u f a c t u r e d b y M o d e r n a u n d e r t h e n a m e S p i k e v a x ® . A d 2 6 . C O V 2 . S r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y J a n s s e n . Th e p r i m a r y s e r i e s f o r A d 2 6 . C O V 2 . S i s o n e d o s e . C h A d O x 1 -S r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y O x f o r d -As t r a Z e n e c a . C h A d O x 1 -S ap p e a r s i n t h i s t a b l e b e c a u s e i t p r o v i d e s f o r s u p p o r t f o r C o n c l u s i o n 3 -3. T h e p r i m a r y s e r i e s f o r A d 2 6 . C O V 2 . S i s o n e d o s e . K e h e t a l . ( 2 0 2 3 ) r e fe r s t o BN T 1 6 2 b 2 a s T o z i n a m e r a n ( P f i z e r ) . N u m b e r o f e v e n t s r e f e r s t o e v e n t s i n v a c c i n e e s o n l y . E M R : e l e c t r o n i c m e d i c a l r e c o r d ; IR R : i n c i d e n c e r a t e r a t i o ; N/ A : n o t a p p l i c a b l e ; RI : r e l a t i v e i n c i d e n c e ; RR : r a t e r a t i o ; SI R : st a n d a r d i z e d i n c i d e n c e r a t i o ; VS D : V a c c i n e S a f e t y D a t a l i n k . SO U R C E S : H a n s o n e t a l . , 2 0 2 2 ; K e h e t a l . , 2 0 2 3 ; K l e i n e t a l . , 2 0 2 1 ; L i e t a l . , 2 0 2 2 ; M o r c i a n o e t a l . , 2 0 2 3 ; P a t o n e e t a l . , 2 0 21 ; S t u r k e n b o o m e t a l . , 20 2 2 ; W a l k e r e t a l . , 2 0 2 2 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 63 PREPUBLICATION COPY—Uncorrected Proofs Keh et al. (2023) retrospectively analyzed data from the National Immunoglobulin Database linked to the National Immunisation Management System, which records all intravenous immunoglobulin (IVIg) prescriptions for GBS patients in England (IVIg is given to an estimated 86 percent of UK patients with GBS). IVIg approval requires adjudication by an independent physician panel (Keh et al., 2023). The study included 11.5 million doses of BNT162b2 2 and 300,000 doses of mRNA-1273.3 Of 196 postvaccinal cases, 21 occurred with BNT162b2 and one with mRNA-1273. Using case numbers from days 43–84 after first-dose vaccination as a comparison group, the first 42 days postvaccination with BNT162b2 had no excess risk of GBS (Keh et al., 2023). Patone et al. (2021) investigated the association between BNT162b2 and GBS among 32.6 million vaccinees, 12.1 million of whom received BNT162b2. This retrospective self-controlled cohort study compared the incidence rate of GBS in England at several intervals (1–7, 8–14, 15– 21, 22–28, and 1–28 days after vaccination) with the rate of GBS during periods outside of this interval. GBS was defined using International Classification of Diseases 10 (ICD-10) codes and identified as the first hospital admission or as a cause of death recorded on the death certificate. Vaccination status was identified in the English National Immunisation Database of COVID-19 vaccination. Only 34 cases of GBS were observed for BNT162b2 during the risk interval. The study found no association between BNT162b2 and GBS at any interval, including the 1–28-day period; incidence rate ratio (IRR) 0.86 (95% confidence interval [CI]: 0.54–1.36) (Patone et al., 2021). The results do not suggest increased incidence, but the estimate is imprecise; the results are consistent with no association but could also be consistent with a small increased risk (Patone et al., 2021). Klein et al. (2021) conducted a surveillance study within the Vaccine Safety Datalink (VSD), which includes data from eight U.S. integrated health care organizations with electronic health records. They compared incidence of GBS among vaccine recipients 1–21 days after either dose 1 or 2 of a messenger ribonucleic acid (mRNA) vaccine with that of concurrent comparators who, on the same calendar day, had received their most recent dose 22–42 days earlier. After 11.8 million doses (57 percent BNT162b2), 10 GBS cases were identified in the risk interval compared with six in the controlled interval, RR 0.70 (95% CI: 0.22–2.31) (Klein et al., 2021). Few events were observed, so the authors were unable to precisely estimate the measure of association. The results would be consistent with no association but could also be consistent with a small increase in risk. Hanson et al. (2022) also analyzed data from VSD. In their primary analysis, they compared the incidence of GBS cases among vaccine recipients at two time intervals, 1–21 and 1– 42 days with that of vaccinated concurrent comparators, who, on the same calendar day, had received their most recent dose 22–42 and 43–84 days earlier, respectively. In addition, incidence of GBS for individual vaccines was compared to prepandemic historical background rate (Hanson et al., 2022). GBS cases were physician adjudicated according to Brighton Collaboration criteria (Sejvar et al., 2011), and the analysis included Brighton Criteria 1–4. Level 1 has the highest level of diagnostic certainty; Level 4 includes suspected cases. The study included 14.6 million doses of mRNA vaccines (BNT162b2 or mRNA-1273) and 483,053 doses of Ad26.COV2.S.4 During the 1–84 days following mRNA vaccines, 36 cases of GBS were confirmed, with nine cases meeting Brighton criteria 1–3 in the 1–21 days risk period. Eleven cases of GBS were confirmed 1–84 days 2 Refers to the COVID-19 vaccine manufactured by Pfizer-BioNTech under the name Comirnaty®. 3 Refers to the COVID-19 vaccine manufactured by Moderna under the name Spikevax®. 4 Refers to the COVID-19 vaccine manufactured by Janssen. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 64 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs after Ad.26.COV2.S, with eight cases meeting Brighton criteria 1–3 in the 1–21 days period. Scan statistics identified days 1–14 after vaccination as a statistically significant cluster (p = .003). In a comparison of Ad26.COV2.S and mRNA vaccines, the adjusted rate ratio in the 1–21 days risk period was 20.56 (95% CI: 6.94–64.66) (Hanson et al., 2022). No association appeared between GBS and any of the vaccines based on the comparison with unvaccinated comparators (Hanson et al., 2022). However, the unadjusted incidence rate at 1–21 and 1–42 days after Ad26.COV2.S was higher than the historical background rate (p < .001). Excluding Brighton Level 4 cases did not significantly alter results. Sturkenboom et al. (2022) conducted a cross-national multi-database retrospective dynamic cohort study using primary and/or secondary health care data from four European countries: Italy, the Netherlands, the United Kingdom, and Spain. They compared the incidence of GBS in vaccine recipients with nonvaccinated persons in 2020 within 28 days after each dose. Of 25.7 million people, 16 GBS cases were identified after BNT162b2, two after Ad26.COV2.S, and none after mRNA-1273. They found an increased risk of GBS 28 days after Ad26.COV2.S (IRR 5.65, 95% CI: 1.40–22.83), but no increased risk after BNT162b2 (IRR 1.10, 95% CI: 0.56–2.15). Results for BNT162b2 suggest no association, but the authors were unable to precisely estimate risk, and results could also be consistent with a small increase in risk (Sturkenboom et al., 2022). Walker et al. (2022) analyzed primary care data from over 17 million patients in England linked to emergency care, hospital admission, and mortality records in OpenSAFELY, which is a secure analytics platform for the National Health Service electronic health records. They used a self-controlled case-series analytical approach where the risk interval was 4–28 days after vaccination. Among 5.7 million recipients of BNT162b2, 283 GBS cases were identified during the risk and controlled intervals; none were identified among 255,446 recipients of mRNA-1273. The results from the study suggested no association between the first dose of BNT162b2 and GBS, although the measure was imprecise and could suggest a small increase in risk (IRR 1.09, 95% CI: 0.75–1.57). Adjusting for calendar time and history of COVID-19 infection did not significantly change the measure of association (IRR 1.00, 95% CI: 0.61–1.64). Li et al. (2022) compared rates of GBS identified through medical records among vaccinees with historical background rates. They used the Clinical Practice Research Datalink (CPRD) AURUM, which contains routinely collected data from UK primary care practices and Spain’s Information System for Research in Primary Care (SIDIAP), a primary care database that covers 80 percent of the population in Catalonia. The study included 3.6 million people who received BNT162b2, 244,913 who received mRNA-1273, 120,731 who received Ad26.CoV.2.S, and 14.3 million people from the general population (Li et al., 2022). Of the BNT162b2 vaccinees, <5 cases occurred within 1–21 days after a first and second dose in CPRD AURUM, compared with 10.4 and 9 expected. SIDIAP showed five cases after the first dose of BNT162b2 and <5 cases after the second dose, compared with 6.3 and 5.3 expected, respectively. For mRNA-1273, <5 cases were diagnosed after the second dose compared with 0.7 expected. No cases were observed with the first dose of mRNA-1273 or after Ad26.COV2.S (Li et al., 2022). Morciano et al. (2023) investigated the association between COVID-19 vaccines and GBS in the population older than 12 years using a self-controlled case-series design with data from several regional health care databases in Italy. They evaluated relative incidence (RI) of GBS during a risk interval of 0–42 days after vaccination and an unexposed interval defined as any time of observation before, between, or after the risk intervals. Of 1.7 million individuals who received mRNA-1273, 25 developed GBS during the study period, with seven and five cases observed with the first and second doses, respectively, during the risk interval (RI 6.83, 95% CI: 2.14–21.85 for Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 65 PREPUBLICATION COPY—Uncorrected Proofs dose 1 and RI 7.41, 95% CI: 2.35–23.38 for dose 2) (Morciano et al., 2023). This corresponded with an estimated 0.4 and 0.3 excess number of cases per 100,000 vaccinated for doses 1 and 2, respectively. The RI of GBS was not significantly increased in the 10.8 million and 581,796 individuals who received BNT162b and Ad26.COV2.S (Morciano et al., 2023). Loo et al. (2022) conducted a retrospective case-control study of all patients admitted for acute polyradiculoneuropathy to two UK neuroscience centers between January 1 and June 30, 2021. They compared vaccinees from the preceding 4 weeks to all GBS patients admitted to their centers between 2005 and 2019. A 2.6-fold (95% CI: 1.98–3.51) increase in admissions for GBS was noted during the time frame, compared to the same period in the preceding 3 years. Of 24 GBS patients, 16 were postvaccine, and all but two (one BNT162b, one mRNA-1273) occurred after ChAdOx1-S (Loo et al., 2022). Although some studies relied on physician adjudication for case ascertainment (Hanson et al., 2022; Keh et al., 2023; Loo et al., 2022), others relied on ICD codes from electronic data without chart confirmation. Some GBS cases identified by the ICD codes might not be true cases, which could have biased the measure of association. In addition, some studies used historical cohorts as a comparator group. Several studies have shown that annual GBS incidence decreased during the pandemic, which could have biased the measure of association. Pharmacovigilance and Surveillance Table 3-2 presents five pharmacovigilance studies that contributed to the committee’s assessment based on their size, design, analytic approach, and region surveilled. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 66 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -2 Ph a r m a c o v i g i l a n c e S t u d i e s i n t h e G u i l l a i n -Ba r r é S y n d r o m e E v i d e n c e R e v i e w Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ran g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) Ab a r a e t a l . (2 0 2 3 ) Co h o r t / hi s t o r i c a l ba c k g r o u n d US VA E R S (p h y s i c i a n ad j u d i c a t e d ) BN T 1 6 2 b 2 ≥1 8 ye a r s 26 6 . 9 mi l l i o n do s e s 21 d a y s : 2 0 9 42 d a y s : 2 5 3 O: E < 1 mR N A -12 7 3 20 2 . 8 mi l l i o n do s e s O: E < 1 Ad 2 6 . C O V 2 . S 17 . 9 mi l l i o n do s e s 1–21 d a y s O: E 3 . 7 9 ( 2 . 8 8 –4. 8 8 ) 1–42 d a y s O: E 2 . 3 4 ( 1 . 8 3 –2. 9 4 ) Ga r c i a - Gr i m s h a w e t al . ( 2 0 2 2 ) Co h o r t / hi s t o r i c a l ba c k g r o u n d Me x i c o Me x i c a n Ep i d e m i o l o g ic a l Su r v e i l l a n c e Sy s t e m / EM R (p h y s i c i a n ad j u d i c a t e d ) BN T 1 6 2 b 2 ≥1 8 ye a r s 16 . 6 mi l l i o n do s e s 32 Un a d j u s t e d I n c i d e n c e 1. 9 2 ( 1 . 3 6 – 2. 7 1 ) mR N A - 12 7 3 2. 3 mi l l i o n do s e s 3 Un a d j u s t e d I n c i d e n c e 1. 2 9 ( 0 . 4 4 –3. 8 1 ) Ad 2 6 . C O V 2 . S 1. 0 mi l l i o n do s e s 4 Un a d j u s t e d I n c i d e n c e 3. 8 6 ( 1 . 5 0 –9. 9 3 ) Ch A d O x 1 -S 38 . 5 mi l l i o n do s e s 37 Un a d j u s t e d i n c i d e n c e 0. 9 6 ( 0 . 7 0 –1. 3 2 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . NE U R O L O G I C CO N D I T I O N S 67 PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -2 Co n t i n u e d Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) Ha e t a l . (2 0 2 3 ) Co h o r t So u t h Ko r e a Gy e o n g g i In f e c t i o u s Di s e a s e Co n t r o l Ce n t e r / EM R (p h y s i c i a n ad j u d i c a t e d ) BN T 1 6 2 b , mR N A -12 7 3 , Ad 2 6 . C O V 2 . S ≥1 2 ye a r s 38 . 8 mi l l i o n do s e s mR N A va c c i n e s : 2 6 ca s e s Ad e n o v i r u s - ve c t o r e d va c c i n e s : 2 9 ca s e s mR N A va c c i n e s I R 0. 8 0 p e r m i l l i o n d o s e s (0 . 4 9 –1. 1 1 ) Ad e n o v i r u s -ve c t o r e d I R 4. 4 9 p e r m i l l i o n d o s e s (2 . 8 5 –6. 1 2 ) Pe g a t e t a l . (2 0 2 1 ) Co h o r t / m R NA va c c i n e s t o Ad e n o v i r u s - ve c t o r e d va c c i n e s US , U K , Eu r o p e Vi g i B a s e (p h y s i c i a n ad j u d i c a t e d ) BN T 1 6 2 b , mR N A -12 7 3 , Ad 2 6 . C O V 2 . S , Ch A d O x 1 No t st a t e d 48 8 m R N A va c c i n e s va c c i n e e s 78 8 Ad e n o v i r u s -ve c t o r e d va c c i n e s va c c i n e e s 14 2 / 1 , 2 5 6 of G B S c a s e s wi t h f a c i a l pa r e s i s ( 2 6 mR N A va c c i n e s , 28 ad e n o v i r u s - ve c t o r e d ) Fa c i a l p a r e s i s m o r e f r e q u e n t wi t h a d e n o v i r u s v e c t o r e d va c c i n e s Ta k u v a e t al . ( 2 0 2 1 ) Op e n -la b e l ph a s e 3 b im p l e m e n t a ti o n s t u d y / hi s t o r i c a l ba c k g r o u n d So u t h Af r i c a Na t i o n a l El e c t r o n i c Va c c i n a t i o n Da t a Sy s t e m / E M R ( p h y s i c i a n ad j u d i c a t e d ) Ad 2 6 . C O V 2 . S ≥1 8 ye a r s 47 7 , 2 3 4 va c c i n e e s 4 c a s e s O: E 5 . 0 9 (1 . 3 9 –13 . 0 2 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 68 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -2 Co n t i n u e d NO T E S : B N T 1 6 2 b 2 r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y P f i z e r -Bi o N T e c h u n d e r t h e n a m e C o m i r n a t y ® . m R N A -12 7 3 r e f e r s t o t h e CO V I D -19 v a c c i n e ma n u f a c t u r e d b y M o d e r n a u n d e r t h e n a m e S p i k e v a x ® . A d 2 6 . C O V 2 . S r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y J a n s s e n . Ch A d O x 1 -S r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y O x f o r d -As t r a Z e n e c a . C h A d O x 1 -S a p p e a r s i n t h i s t a b l e b e c a u s e i t p r o v i d e s f o r s u p p o rt fo r C o n c l u s i o n 3 -3. H a e t a l . ( 2 0 2 3 ) c o m b i n e d t h e n u m b e r o f e v e n t s f r o m a d e n o v i r a l v e c t o r v a c c i n e s ( A d 2 6 . C O V 2 . S a n d C h A d O x 1 -S) . C I : co n f i d e n c e i n t e r v a l ; E M R : e l e c t r o n i c m e d i c a l r e c o r d ; I R : i n c i d e n c e r a t e ; O : E : o b s e r v e d t o e x p e c t e d r a t i o ; V A E R S : V a c c i n e Ad v e r s e E v e n t s Re p o r t i n g S y s t e m . SO U R C E S : A b a r a e t a l . , 2 0 2 3 ; G a r c í a -Gr i m s h a w e t a l . , 2 0 2 2; Ha e t a l . , 2 0 2 3 ; Pe g a t e t a l . , 2 0 2 2 ; T a k u v a e t a l . , 2 0 2 2. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 69 PREPUBLICATION COPY—Uncorrected Proofs Abara et al. (2023) analyzed data from the Vaccine Adverse Event Reporting System, which is comanaged by the Centers for Disease Control and Prevention and Food and Drug Administration. Of 487.7 million COVID-19 vaccine doses, 209 and 253 reports of GBS occurred within 21 and 42 days, respectively. Observed-to-expected ratios (O:E) were 3.79 (95% CI: 2.88–4.88) for days 1–21 and 2.34 (95% CI: 1.83–2.94) for days 1–42 after Ad26.COV2.S and less than 1 (not significantly increased) after BNT162b2 and mRNA-1273 for both postvaccination periods (Abara et al., 2023). Pegat et al. (2022) analyzed data from VigiBase, the World Health Organization pharmacovigilance database, and the French pharmacovigilance database to compare the frequency of facial paralysis in GBS cases after adenovirus-vector (AV) vaccines to that after mRNA vaccines and found that 142 of 1,256 GBS patients in VigiBase had associated facial paralysis (11.3 percent). This included 26 of 488 who received mRNA vaccines (12/328 BNT162b2, 14/160 mRNA-1273), 114 of 744 who received AV vaccines (28/114 Ad26.COV2.S, 86/630 ChAdOx1-S), and 2 of 24 who received other vaccines. Facial paralysis was significantly more frequent after AV vaccines (χ2: p = 6.44 × 10−8) (Pegat et al., 2022). García-Grimshaw et al. (2022) conducted a retrospective analysis of a nationwide passive registry of GBS among recipients of 81.8 million doses of seven COVID-19 vaccines in Mexico. The overall observed incidence was 1.19 per 1 million doses (95% CI: 0.97–1.45), which was higher for Ad26.COV2.S (3.86 per 1 million doses, 95% CI: 1.50–9.93) and BNT162b2 (1.92 per 1 million doses, 95% CI: 1.36–2.71) (García-Grimshaw et al., 2022). Ha et al. (2023) conducted a prospective regional surveillance study for GBS in the Gyeonggi Province, South Korea. Out of 38.8 million vaccine doses, 55 cases of physician adjudicated GBS were identified. The incidence rate of GBS after AV vaccines (Ad26.COV2.S, ChAdOx1-S) was 4.49 per million doses (95% CI: 2.85–6.12), compared to 0.80 per million doses after mRNA vaccines (BNT162b2, mRNA-1273) (95% CI: 0.49–1.11) (Ha et al., 2023). Takuva et al. (2022) evaluated the incidence rate of GBS in all health care workers in South Africa registered in the national Electronic Vaccination Data System after receiving Ad26.COV2.S. Four cases of GBS were recorded, with an observed-to-expected ratio of 5.09 (95% CI: 1.39–13.02) (Takuva et al., 2022). From Evidence to Conclusions The totality of the evidence included several large studies that minimized confounding bias by using self-controlled or concurrent cohort design or by relying on chart review for case ascertainment; none of the epidemiological studies reported a significant risk of GBS after BNT162b2. This is reinforced by the pharmacovigilance data; although they were more prone to confounding bias, multiple large studies surveilling different population cohorts worldwide consistently identified an increased risk with AV but not mRNA vaccines despite potential differing coding trends, seasonality, co-infections, and co-administration of other vaccines. Conclusion 3-1: The evidence favors rejection of a causal relationship between the BNT162b2 vaccine and Guillain-Barré syndrome. In general, relatively few mRNA-1273 doses were included in the studies. Only one study reported an increased risk of GBS after the first and second dose, although the CIs for the measure of association were very wide (Morciano et al., 2023). The study also reported that the Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 70 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs excess number of cases was very small (<1 case per 100,000 doses). Morciano et al. (2023) was the only study to utilize the relatively longer risk period of 0–42 days without relying on chart review for case ascertainment. Although the study used a self-controlled strategy to minimize bias, its reliance on ICD codes combined with the prolonged risk interval may have led to inclusion of some historical cases rather than true incident cases. Two other studies included a larger number of vaccines and used a vaccinated concurrent cohort design (Hanson et al., 2022; Klein et al., 2021). As noted, the pharmacovigilance data also favored lack of an association between GBS and the mRNA vaccines, and the platforms used in mRNA-1273 and BNT162b2 are similar. Additionally, strong mechanistic evidence linking mRNA vaccines to GBS is lacking. Conclusion 3-2: The evidence favors rejection of a causal relationship between the mRNA-1273 vaccine and Guillain-Barré syndrome. Four epidemiology studies included patients who received Ad26.COV2.S. One study found an increased risk of GBS compared to a historical cohort, even though it did not find an association in its primary analysis, which used a vaccinated concurrent cohort design (Hanson et al., 2022). Unlike other studies reviewed, cases were physician adjudicated according to Brighton Criteria, and the increased risk was still observed when Level 4 cases (suspected GBS) were excluded. Although the analysis included two risk periods, 1–21 and 1–42 days, the vast majority of cases occurred in the first period, which is in keeping with expected latency based on historical precedent and presumed mechanism. Sturkenboom et al. (2022) also found an increased risk when comparing Ad26.COV2.S recipients with a 2020 cohort of unvaccinated individuals, although the total number of events was small and the CI wide. No association was observed in the other two studies (Li et al., 2022; Morciano et al., 2023). Li et al. had a comparatively low number of vaccinees. Although ChAdOx1-S was not formally within the purview of the committee, five of the studies observed an increased risk of GBS (Keh et al., 2023; Loo et al., 2022; Morciano et al., 2023; Patone et al., 2021; Walker et al., 2022). These included studies with a large number of participants and designs that minimize confounding bias. Additionally, two studies reported a higher rate of the facial paresis variant in patients who received either AV vaccine compared to historical cohorts (Hanson et al., 2022; Loo et al., 2022). This trend was not observed in Keh et al. (2023) despite reporting an increased risk of GBS after ChAdOx1-S. Evidence from pharmacovigilance databases spanning different regions worldwide also documented an increased risk with the AV vaccines, and one study (Pegat et al., 2022) observed an increased rate of facial paresis associated with AV but not mRNA vaccines. The epidemiological association between GBS and ChAdOx1-S but not mRNA vaccines suggests that the mechanism is unlikely to relate to immune responses to the spike protein itself. In addition, the reported increased rates of a rare variant (facial paresis) after vaccination with both related, albeit not identical, AV vaccines suggest a potential shared mechanism, although no definitive one was identified by the committee in the mechanistic literature, and this pattern was not observed in all studies. Differences in the AV platforms and their respective receptor, however, should give pause when extrapolating from one such vaccine to another. The totality of evidence for Ad26.COV2.S includes two well-designed, positive epidemiological studies and pharmacovigilance data, strong supporting epidemiological evidence Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 71 PREPUBLICATION COPY—Uncorrected Proofs from ChAdOx1-S, and the potential for a platform-specific mechanism in both AV vaccines. No epidemiological literature evaluated the relationship between NVX-CoV2373 5 and GBS. Conclusion 3-3: The evidence favors acceptance of a causal relationship between the Ad26.COV2.S vaccine and Guillain-Barré syndrome. Conclusion 3-4: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and Guillain-Barré syndrome. 5 Refers to the COVID-19 vaccine manufactured by Novavax. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 72 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs CHRONIC INFLAMMATORY DEMYELINATING POLYNEUROPATHY BOX 3-2 Conclusions for Chronic Inflammatory Demyelinating Polyneuropathy Conclusion 3-5: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and chronic inflammatory demyelinating polyneuropathy. Conclusion 3-6: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and chronic inflammatory demyelinating polyneuropathy. Conclusion 3-7: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and chronic inflammatory demyelinating polyneuropathy. Conclusion 3-8: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and chronic inflammatory demyelinating polyneuropathy. Background Chronic inflammatory demyelinating polyneuropathy (CIDP), also known as “chronic inflammatory demyelinating polyradiculoneuropathy,” is an acquired, immune-mediated disorder affecting the peripheral nerve and roots. As with GBS, CIDP is now considered a group of disorders all sharing clinical and electrodiagnostic features but with probable heterogenous underlying mechanisms. Typical CIDP, the most prevalent CIDP variant, accounts for 50–60 percent of cases, presents as relapsing-remitting or gradually progressive symmetric limb weakness over a period of months. Sensory loss is common, and deep tendon reflexes are absent or reduced. Cranial nerve involvement occurs in 10–20 percent of cases. Acute onset resembling GBS can occur in 5–16 percent of cases but, unlike GBS, where symptom progression ends within 4 weeks, symptoms continue to progress beyond 8 weeks (McCombe et al., 1987; Thomas et al., 1987) (a minimum of 2 months of symptoms is required to make the diagnosis per CIDP diagnostic criteria; Van den Bergh et al., 2010). The reported incidence of CIDP is 0.3–1.6 cases per 100,000 person-years (Laughlin et al., 2009), with a male predominance and incidence rising with advancing age and some studies reporting a mean age at presentation of 60 years (Hafsteinsdottir and Olafsson, 2016). Electrodiagnostic evidence of nerve demyelination and elevated CSF protein with a normal leukocyte count supports the diagnosis. A nerve biopsy demonstrating segmental demyelination with or without inflammation can be diagnostic but is rarely needed. CIDP variants are recognized, and their distinctive clinical characteristics are included in European Academy of Neurology/Peripheral Nerve Society diagnostic criteria (Van den Bergh et al., 2021). These include typical, distal (or distal acquired demyelinating distal neuropathy), multifocal (or multifocal acquired demyelinating sensory and motor neuropathy), focal, motor, and sensory CIDP (Van den Bergh et al., 2021). Definitions of what constitute CIDP continue to evolve, and certain conditions classed as CIDP variants in the past, including chronic immune sensory Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 73 PREPUBLICATION COPY—Uncorrected Proofs polyradiculopathy and the autoimmune paranodopathies, were excluded from the most recent criteria because the underlying nerve injury is not definitively demyelinating. Mechanisms Although the pathophysiology of CIDP and its variants is not known, evidence supports an immune-mediated mechanism as the main cause. Characteristic features include segmental demyelination and remyelination and varying degrees of endoneurial macrophage infiltration (Dalakas, 2011). Levels of T helper 17 cells are increased in the peripheral blood and CSF, as are levels of soluble adhesion molecules, chemokines, and metalloproteinases (Dalakas, 2011). The apparent effectiveness of plasmapheresis, which purportedly removes pathogenic antibodies along with other inflammatory mediators, suggests that circulating humoral factors and autoantibodies may be involved. Complement fixation on the myelin sheath of nerves of some with CIDP also suggests a potential antibody-mediated mechanism (Dalakas and Engel, 1980). Antibodies directed against nodal and paranodal proteins, such as contactin-1, and neurofascin isoforms, are found in a subset of patients with clinical features suggestive of CIDP. However, nerve biopsies in these patients do not show the distinctive features of CIDP, and this is now considered a separate entity (autoimmune paranodopathies) (Van den Bergh et al., 2021). One study identified potentially cross-reactive epitopes shared between the SARS-CoV-2 spike protein and neuronal structures using a bioinformatics approach (Felipe Cuspoca et al., 2022), suggesting that molecular mimicry as a cause of potential neurological harms of COVID- 19 vaccines is plausible, but evidence supporting this hypothesis is lacking. Epidemiological Evidence Clinical trial results submitted to FDA for Emergency Use Authorization and/or full approval do not indicate a signal regarding CIDP and any of the vaccines under study (FDA, 2021, 2023a, 2023b, 2023c). Table 3-3 summarizes one study that contributed to the causality assessment. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 74 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -3 Ep i d e m i o l o g i c a l S t u d y i n t h e C h r o n i c I n f l a m m a t o r y D e m y e l i n a t i n g P o l y r a d i c u l o n e u r o p a t h y E v i d e n c e R e v i e w Au t h o r St u d y D e s i g n an d C o n t r o l Gr o u p Lo c a t i o n Da t a So u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r of E v e n t s Re s u l t s Lo o e t a l . (2 0 2 1 ) Ca s e -co n t r o l st u d y / h i s t o r i c a l ba c k g r o u n d UK EM R (p h y s i c i a n ad j u d i c a t e d ) BN T 1 6 2 b 2 ≥1 6 ye a r s 24 p a t i e n t s ad m i t t e d wi t h a c u t e o n s e t po l y r a d i c u l o - ne u r o p a t h y be t w e e n Ja n u a r y –Ju n e 20 2 1 1 4 c a s e s re c l a s s i f i e d as a c u t e on s e t C I D P ; No c a s e s fo l l o w e d mR N A va c c i n e s mR N A -12 7 3 1 NO T E S : B N T 1 6 2 b 2 r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y P f i z e r -Bi o N T e c h u n d e r t h e n a m e C o m i r n a t y ® . m R N A -12 7 3 re f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y M o d e r n a u n d e r t h e n a m e S p i k e v a x ® . N u m b e r o f e v e n t s r e f e r s t o e v e n t s i n v a c c i n e e s o n l y . EM R : e le c t r o n i c m e d i c a l r e c o r d . SO U R C E : L o o e t a l . , 2 0 2 2 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 75 PREPUBLICATION COPY—Uncorrected Proofs Loo et al. (2021) conducted a retrospective case-control study of all patients admitted with acute-onset polyradiculoneuropathy to two UK neuroscience centers, January 1–June 30, 2021. Of 24 GBS patients, 16 were postvaccination and all but two (1 BNT162b, 1 mRNA-1273) were after ChAdOx1-S. Four cases initially classified as GBS were eventually reclassified as acute-onset CIDP due to progression or relapse past 8 weeks from onset; all four had received ChAdOx1-S.6 From Evidence to Conclusions Epidemiological and mechanistic evidence are absent. Only one small case-control study evaluated the association between COVID-19 vaccines and CIDP; four cases initially classified as GBS were later reclassified as acute-onset CIDP, and no historical background rate was offered for comparison. Conclusion 3-5: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and chronic inflammatory demyelinating polyneuropathy. Conclusion 3-6: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1723 vaccine and chronic inflammatory demyelinating polyneuropathy. Conclusion 3-7: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and chronic inflammatory demyelinating polyneuropathy. Conclusion 3-8: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and chronic inflammatory demyelinating polyneuropathy. 6 ChAdOx1-S refers to the COVID-19 vaccine manufactured by Oxford-AstraZeneca. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 76 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs BELL’S PALSY BOX 3-3 Conclusions for Bell’s Palsy Conclusion 3-9: The evidence favors rejection of a causal relationship between the BNT162b2 vaccine and Bell’s Palsy. Conclusion 3-10: The evidence favors rejection of a causal relationship between the mRNA-1273 vaccine and Bell’s Palsy. Conclusion 3-11: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and Bell’s Palsy. Conclusion 3-12: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and Bell’s Palsy. Background BP is an idiopathic, unilateral, self-limited, acute facial nerve paresis or paralysis. It occurs with equal frequency on either side of the face and usually resolves within weeks or months. It can lead to severe temporary oral insufficiency and an incapability to close the eyelids, resulting in potentially permanent eye injury. In approximately 25 percent of patients, moderate-to-severe facial asymmetry may persist and affect quality of life (Zhang et al., 2020b). BP is the most common acute mononeuropathy (Zhang et al., 2020b), with an incidence of 11.5–53.3 per 100,000 person-years (Baugh et al., 2013). It is estimated that every year, about 40,000 U.S. people are affected (NORD, 2022). The risk factors are poorly understood. Risk may increase with age, but no indication exists that one sex or geographical area is more at risk (Kim and Park, 2021). BP symptoms typically develop quickly, with maximum symptoms occurring within 72 hours (W. Zhang et al., 2020). Mechanisms The etiology of BP is unknown, but theories fall into five categories: anatomical, viral, ischemic, inflammatory, and due to cold exposure (based on season or local climate) (W. Zhang et al., 2020). When considering the possibility of a vaccine trigger of BP, it is unlikely that anatomy, ischemia, or cold stimulation would play a role. Evidence supporting inflammation includes demonstrated gadolinium enhancement of the facial nerve on MRI of the brain and CSF pleocytosis in many patients with BP (Steiner and Mattan, 1999). Histopathology from one autopsy study demonstrated a lymphohistiocytic infiltrate within all layers of the nerve and inflammation that extended to the geniculate ganglion but spared most ganglion cells (Liston and Kleid, 1989). Infection may be a cause of BP. Infectious facial palsy has been most clearly linked to Borrelia burgdorferi (the bacteria that causes Lyme disease), and varicella zoster virus reactivation (Ramsay Hunt syndrome). Many have argued for a link between herpes simplex virus type 1 (HSV-1) reactivation and BP (W. Zhang et al., 2020), and acyclovir is routinely Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 77 PREPUBLICATION COPY—Uncorrected Proofs prescribed to patients with BP. Arguments against a pathophysiological role for HSV-1 include that it resides in the peripheral sensory ganglia and reactivation is not associated with motor weakness, that it tends to recur, whereas BP tends to be monophasic, and that HSV-1 outbreaks are common, whereas BP is rare (Steiner and Mattan, 1999). Finally, in a randomized controlled trial with a factorial design in which patients received 10 days of prednisolone, acyclovir, both, and placebo, prednisolone significantly improved outcomes, whereas acyclovir did not (Sullivan et al., 2007). Infection may also cause BP via a post-infectious immune-mediated mechanism rather than by direct invasion of the nerve. Such mechanisms could include bystander activation, epitope spreading or polyclonal activation of previously dormant self-reactive lymphocytes (Chapter 2). Arguments favoring an infectious trigger of BP include that it can occur in epidemic clusters (Leibowitz, 1969) and displays seasonal variation (Kim and Park, 2021). Potential triggers include cytomegalovirus, Epstein-Barr virus, mumps, rubella, and HIV (Steiner and Mattan, 1999). An intranasal influenza vaccine which has since been removed from the market was associated with BP (Wratten et al., 1977). In this case-control study, BP most often occurred within 31-60 days following vaccination arguing against a direct toxic effect and in favor of an immune-mediated mechanism. Recent evidence has suggested a possible association between COVID-19 infection and BP (Rafati et al., 2023). The fact that there are multiple putative viral triggers argues against molecular mimicry as a mechanism. Patients with BP have been shown to have elevated levels of the cytokines IL-6, IL-8, and TNF-alpha compared to controls (Yılmaz et al., 2002). Some have argued that cytokine- mediated neuronal damage, in particular by type 1 interferon (type 1 IFN), might mediate neurological adverse events after COVID-19 vaccination (Chen et al., 2022a; Shemer et al., 2021). Because BP has been seen as a complication of type 1 IFN treatment for hepatitis C (Hwang et al., 2004), some have postulated that an elevation of type 1 IFN after COVID-19 vaccination could be associated with it (Shemer et al., 2021). Single-cell transcriptomics demonstrate a strong interferon signature after booster mRNA vaccination (Arunachalam et al., 2021), but this has not been correlated with neurological harms. Adenoviral vaccines have also been shown to induce an interferon signature, at least in mice (Sheerin et al., 2021). However, no studies link cytokine responses after vaccination to neurological events. Epidemiological Evidence Clinical trial results submitted to FDA for Emergency Use Authorization and/or full approval do not indicate a signal regarding Bell’s Palsy and any of the vaccines under study (FDA, 2021, 2023a, 2023b, 2023c). Table 3-4 presents 11 studies that contributed to the causality assessment. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 78 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -4 Ep i d e m i o l o g i c a l St u d i e s i n t h e B e l l ’ s P a l s y E v i d e n c e R e v i e w Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a So u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r of E v e n t s Re s u l t s (9 5 % C I ) Ab Ra h m a n et a l . (2 0 2 2 ) Se l f - co n t r o l l e d ca s e s e r i e s Ma l a y s i a EM R BN T 1 6 2 b 2 ≥1 2 y e a r s Do s e 1 : 8. 7 mi l l i o n va c c i n e e s Do s e 1 : 1 7 Do s e 1 : IR R 1 . 3 2 ( 0 . 7 7 – 2. 2 4 ) Do s e 2 : 6. 7 mi l l i o n va c c i n e e s Do s e 2 : 1 0 Do s e 2 : IR R 0 . 8 8 ( 0 . 4 5 – 1. 7 3 ) Kl e i n et al . (2 0 2 1 ) Co h o r t w i t h va c c i n a t e d co n c u r r e n t co m p a r a t o r s US EM R BN T 1 6 2 b 2 ≥1 6 y e a r s 6. 8 m i l l i o n do s e s BN T 1 2 b 2 an d mR N A - 12 7 3 co m b i n e d : 53 5 BN T 1 2 b 2 a n d mR N A -12 7 3 co m b i n e d : RR 1 . 0 0 ( 0 . 8 6 –1. 1 7 ) mR N A - 12 7 3 5. 1 mi l l i o n do s e s Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . NE U R O L O G I C CO N D I T I O N S 79 PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s Li e t a l . (2 0 2 2 ) Co h o r t a n d se l f - co n t r o l l e d / B a ck g r o u n d ra t e s a n d se l f - co n t r o l l e d UK a n d Sp a i n EM R Cl i n i c a l Pr a c t i c e Re s e a r c h Da t a l i n k AU R U M da t a b a s e (U K ) In f o r m a t i o n S y s t e m fo r Re s e a r c h i n Pr i m a r y Ca r e (S I D I A P ) da t a b a s e (S p a i n ) BN T 1 6 2 b 2 (U K a n d Sp a i n ) >18 y e a r s UK Do s e 1 : 1. 7 mi l l i o n va c c i n e e s Do s e 2 : 1. 2 mi l l i o n va c c i n e e s UK Do s e 1 : 4 6 Do s e 2 : 2 4 UK Do s e 1 : SI R 0 . 4 0 ( 0 . 3 0 –0. 5 3 ) Do s e 2 : SI R 0 . 2 4 ( 0 . 1 6 –0. 3 6 ) SC C S IR R 0 . 8 3 ( 0 . 6 6 –1. 0 2 ) Sp a i n Do s e 1 : 1. 9 mi l l i o n va c c i n e e s Do s e 2 : 1. 3 mi l l i o n va c c i n e e s Sp a i n Do s e 1 : 1 0 0 Do s e 2 : 8 5 Sp a i n Do s e 1 : SI R 0 . 8 6 ( 0 . 7 0 – 1. 0 4 ) Do s e 2 : SI R 0 . 8 8 ( 0 . 7 1 –1. 0 8 ) SC C S : IR R 0 . 8 3 ( 0 . 6 6 –1. 0 2 ) mR N A - 12 7 3 (S p a i n on l y ) Do s e 1 : 24 4 , 9 1 3 va c c i n e e s Do s e 2 : 16 0 , 2 2 8 va c c i n e e s Do s e 1 : 1 4 Do s e 2 : 5 Do s e 1 : SI R 0 . 9 2 ( 0 . 5 4 –1. 5 5 ) Do s e 2 : SI R 0 . 4 4 ( 0 . 1 8 –1. 0 6 ) SC C S : IR R 0 . 9 9 ( 0 . 5 4 –1. 6 4 ) Ad 2 6 . C O V 2. S (S p a i n on l y ) Do s e 1 : 12 0 , 7 3 1 va c c i n e e s Do s e 1 : 6 SI R 1 . 1 5 ( 0 . 5 2 –2. 5 6 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 80 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -4 Co n t i n u e d Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a So u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r of E v e n t s Re s u l t s (9 5 % C I ) Pa t o n e e t al . ( 2 0 2 1 ) Se l f - co n t r o l l e d ca s e s e r i e s UK EM R BN T 1 6 2 b 2 ≥ 1 6 y e a r s Do s e 1 : 12 . 1 mi l l i o n va c c i n e e s 24 7 IR R 1 . 0 6 (0 . 9 0 –1. 2 6 ) Sh a s h a e t al . ( 2 0 2 1 ) Ma t c h e d co h o r t va c c i n a t e d vs . un v a c c i n a t e d Is r a e l EM R BN T 1 6 2 b 2 ≥1 6 y e a r s Do s e 1 : 23 3 , 1 5 9 va c c i n e e s 23 RR 0 . 9 6 ( 0 . 5 4 –1. 7 0 ) Sh e m e r e t al . ( 2 0 2 1 ) Ca s e c o n t r o l Is r a e l Ho s p i t a l i z a -ti o n d a t a BN T 1 6 2 b 2 50 . 9 + 20 . 2 ye a r s 37 ca s e s 21 OR 0 . 8 4 ( 0 . 3 7 –1. 9 0 ) Sh i b l i e t al . ( 2 0 2 1 ) Co h o r t u s i n g ba c k g r o u n d ra t e Is r a e l EM R BN T 1 6 2 b 2 ≥1 6 y e a r s Do s e 1 : 2. 6 mi l l i o n va c c i n e e s 13 2 SI R 1 . 3 6 ( 1 . 1 4 –1. 6 1 ) Do s e 2 : 2. 4 mi l l i o n va c c i n e e s 15 2 SI R 1 . 1 6 ( 0 . 9 9 –1. 3 6 ) Sh o a i b i et al . ( 2 0 2 3 ) Se l f - co n t r o l l e d ca s e s e r i e s US Cl a i m s - ba s e d da t a w i t h me d i c a l re c o r d re v i e w BN T 1 6 2 b 2 (b o o s t e r do s e ) ≥6 5 y e a r s 6. 2 m i l l i o n va c c i n e e s 1, 6 7 4 IR R 1 . 1 3 ( 0 . 7 7 –1. 6 5 ) mR N A - 12 7 3 (b o o s t e r do s e ) 1, 5 9 4 IR R 1 . 0 2 ( 0 . 7 0 –1. 5 0 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . NE U R O L O G I C CO N D I T I O N S 81 PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -4 Co n t i n u e d Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a So u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) St u r k e n - bo o m et a l . (2 0 2 2 ) Co h o r t u s i n g ba c k g r o u n d ra t e It a l y , Ne t h e r l a n ds U K a n d Sp a i n EM R BN T 1 6 2 b 2 Va r i e d 54 % of 1 2 . 1 mi l l i o n va c c i n e e s to t a l 14 9 IR R 0 . 8 7 ( 0 . 6 9 –1. 1 0 ) mR N A - 12 7 3 6% 27 IR R 0 . 9 9 ( 0 . 6 8 –1. 4 5 ) Ad 2 6 . C O V 2. S 2% 6 IR R 1 . 0 8 ( 0 . 4 5 –2. 6 0 ) Ta k e u c h i et a l . (2 0 2 2 ) Co h o r t a n d se l f - co n t r o l l e d ca s e s e r i e s (S C C S ) Ja p a n EM R mR N A (B N T 1 6 2 b 2, m R N A - 12 7 3 ) ≥ 1 8 y e a r s Do s e 1 : 13 6 , 6 6 7 va c c i n e e s Do s e 2 : 12 7 , 3 2 2 va c c i n e e s Co h o r t : Do s e 1 : 1 Do s e 2 : 1 Do s e 1 : IR R 1 . 1 4 ( 0 . 2 7 – 4. 8 9 ) Do s e 2 : IR R 0 . 6 0 ( 0 . 0 8 –4. 4 9 ) SC C S : Do s e 1 : 1 5 Do s e 2 : 1 5 Do s e 1 : IR R 1 . 0 3 ( 0 . 2 0 –5. 3 1 ) Do s e 2 : IR R 0 . 4 7 ( 0 . 0 5 –4. 1 8 ) Wa l k e r e t al . ( 2 0 2 2 ) Se l f - co n t r o l l e d ca s e s e r i e s En g l a n d EM R BN T 1 6 2 b 2 18 –10 5 ye a r s 5, 7 2 9 , 1 5 2 3, 6 0 9 Do s e 1 : IR R 0 . 8 8 ( 0 . 7 6 –1. 0 2 ) Do s e 2 : IR R 0 . 9 2 (0 . 7 8 –1. 1 0 ) mR N A - 12 7 3 25 5 , 4 4 6 78 Do s e 1 : IR R 0 . 8 0 ( 0 . 2 4 –2. 6 2 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 82 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -4 Co n t i n u e d NO T E S : BN T 1 6 2 b 2 r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y P f i z e r -Bi o N T e c h u n d e r t h e n a m e C o m i r n a t y ® . m R N A -12 7 3 r e f e r s t o t h e CO V I D -19 v a c c i n e m a n u f a c t u r e d b y M o d e r n a u n d e r t h e n a m e S p i k e v a x ® . Ad 2 6 . C O V 2 . S r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y Ja n s s e n . Th e p r i m a r y s e r i e s f o r A d 2 6 . C O V 2 . S i s o n e d o s e . N u m b e r o f e v e n t s r e f e r s t o e v e n t s i n v a c c i n e e s o n l y . CI : c o n f i d e n c e i n t e r v a l ; E M R : el e c t r o n i c m e d i c a l r e c o r d ; I R R : i n c i d e n c e r a t e r a t i o ; O R : o d d s r a t i o ; R R : r e l a t i v e r i s k ; S C C S : s e l f -co n t r o l l e d c a s e s e r i e s ; S I R : s t a n d a r d i z e d in c i d e n c e r a t i o . SO U R C E S : Ab R a h m a n e t a l . , 2 0 2 2 ; K l e i n e t a l . , 2 0 2 1 ; L i e t a l . , 2 0 2 2 a ; P a t o n e e t a l . , 2 0 2 1 ; S h a s h a e t a l . , 2 0 2 2 ; S h e m e r e t a l . , 2 0 2 1 ; Sh i b l i e t a l . , 20 2 1 ; S h o a i b i e t a l . , 2 0 2 3 ; S t u r k e n b o o m e t a l . , 2 0 2 2 ; T a k e u c h i e t a l . , 2 0 2 2 ; W a l k e r e t a l . , 2 0 2 2 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 83 PREPUBLICATION COPY—Uncorrected Proofs Patone et al. (2021) investigated the association between BNT162b2 and BP among 12.1 million vaccinees in England using a self-controlled case series (SCCS) study. They compared the incidence rate of BP in the interval of 1–28 days after vaccination with that during periods outside of this interval. BP was defined using ICD-10 codes and identified as the first hospital admission or as a cause of death recorded on the death certificate (Patone et al., 2021). Vaccination status was identified in the English National Immunization Database of COVID-19 vaccination; they identified 250 BP cases and found no association with BNT162b2 (incidence rate ratio (IRR) 1.06 (95 percent confidence interval (CI): 0.80–1.25) (Patone et al., 2021). Walker et al. (2022) analyzed primary care data from more than 17 million patients in England linked to emergency care, hospital admission, and mortality records in the OpenSAFELY platform (Walker et al., 2022). They excluded BP cases that occurred before the study start date. Cases were determined from any primary care, emergency department, hospital admission, or mortality records. They used an SCCS analytical approach where the risk interval was 4–28 days after vaccination. Among 5.7 million recipients of BNT162b2, 3,609 BP cases were identified, and among 255,446 recipients of mRNA-1273, 78 BP cases were identified. They found no association between the first dose of BNT162b2 and BP (IRR 0.89, 95% CI: 0.76–1.03) or the second dose (IRR 0.92, 95% CI: 0.78–1.10). Similarly, no association appeared with mRNA-1273 after the first or second dose (IRR 0.59, 95% CI: 0.13–2.62 and IRR 0.80, 95% CI: 0.24–2.62, respectively) (Walker et al., 2022). Ab Rahman et al. (2022) conducted a self-controlled case-series study among hospitalized BP cases in Malaysia. Vaccination status was determined from the national COVID- 19 register data. The incidence of BP was assessed during a 21-day risk interval after vaccination relative to a control period using conditional Poisson regression with adjustment for calendar time. After more than 15 million doses of BNT162b2, 27 cases of BP were identified in the risk interval. Compared with the control interval, no significant increased risk of BP occurred after the first (IRR 1.32, 95% CI: 0.77–2.24) or second (IRR 0.88, 95% CI: 0.45–1.73) dose. The IRR after any dose was 1.11 (95% CI: 0.77–1.75) (Ab Rahman et al., 2022). Li et al. (2022b) evaluated the association between vaccination and BP using two study designs: a population-based cohort design where they compared rates of BP identified through medical records among vaccinees with historical background rates and an SCCS analysis. They used CPRD AURUM and SIDIAP. The study included 3.6 million people who received BNT162b2, 244,913 who received mRNA-1273, 120,731 who received Ad26.CoV.2, and 14.3 million people from the general population. Of the BNT162b2 vaccinees, 46 and 24 BP cases occurred after a first and second dose in CPRD AURUM, compared with 116.4 and 99.5 expected. The standardized incidence ratio (SIR) was 0.40 (95 percent CI: 0.30–0.53) for the first and 0.24 (95 percent CI: 0.16–0.36) for the second dose. SIDIAP had 100 and 85 BP cases after the first and second dose of BNT162b2, compared with 116.7 and 97.1 expected. SIR was 0.86 (95% CI: 0.70–1.04) for the first and 0.88 (95% CI: 0.71–1.08) for the second dose. For mRNA- 1273, 14 and 5 cases occurred after the first and second dose compared with 15.2 and 11.3 expected. The corresponding SIRs are 0.92 (95% CI: 0.54–1.55) and 0.4 (95% CI: 0.18–1.06). For Ad26.COV2.S, six BP cases were identified compared with 5.2 expected, corresponding to an SIR of 1.15 (95% CI: 0.52–2.56) (Li et al., 2022). The SCCS analysis was only sufficiently powered to study those with a first dose of BNT162b2 and mRNA-1273. In CPRD AURUM, the adjusted IRR of BP 1–21 days after vaccination was 0.83 (95% CI: 0.61–1.10) for BNT162b2. In SIDIAP, the adjusted IRR was 0.83 (95% CI: 0.66–1.02) for BNT162b2 and 0.99 (95% CI: 0.54–1.64) for mRNA-1273 (Li et al., 2022). Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 84 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs Shibli et al. (2021) used data from the computerized database of Clalit Health Services, which provides inclusive health care for more than half of the Israeli population, to assess whether BNT162b2 was associated with increased risk by comparing BP rates in vaccinees with historical rates in the general population. They assessed rates 21 days after the first dose and 30 days after the second dose. Overall, 132 cases of BP were reported in 2.6 million vaccinees with the first dose compared with 97.1 expected, and 152 cases in 2.4 million vaccinees were reported compared with 130.49 expected after the second dose. The age- and sex-weighted SIRs were 1.36 (95% CI: 1.14–1.61) and 1.16 (95% CI: 0.99–1.36) after the first and second doses, respectively. Although more cases were observed than expected, the attributable risk fraction was 0.26 for the first and 0.14 for the second dose. The attributable risk per 100,000 vaccinees was 1.35 for the first and 0.86 for the second dose (Shibli et al., 2021). Shasha et al. (2021) conducted a matched cohort study in which they compared risk of BP in 233,159 BNT162b2 vaccinees with that in 233,159 age- and sex-matched unvaccinated individuals. BP cases were identified by ICD-10 code and confirmed by chart review. Of the 123 cases identified by ICD-10 codes, 76 were excluded because they were not incident cases or not consistent with BP. Vaccinated and unvaccinated individuals had 23 versus 24 cases of BP (RR 0.96, 95% CI: 0.54–1.70). Sturkenboom et al. (2022) conducted a cross-national multi-database retrospective dynamic cohort study using primary and/or secondary health care data from four European countries: Italy, the Netherlands, the United Kingdom, and Spain. Individuals were required to have at least 365 days of data availability before cohort entry. The end of follow-up was the earliest dates of BP occurrence, last data collection, or death. Person-time after the start of the study was divided in two main periods, nonvaccinated and vaccinated; the latter started at the first of any of the COVID-19 vaccine and lasted for a maximum of 28 days after dose 1 and 28 days after dose 2 or until the date of last data available. Of the 25.7 million people included, 149 BP cases were identified after BNT162b2, 27 after mRNA-1273, and 6 after Ad26.COV2.S. They found no increased risk of BP 28 days after BNT162b2 (IRR 0.87, 95% CI: 0.69–1.10), mRNA-1273 (IRR 0.99, 95% CI: 0.68–1.45), or Ad26.COV2.S (IRR 1.08, 95% CI: 0.45–2.60) (Sturkenboom et al., 2022). Shemer et al. (2021) conducted a case-control study using data from the emergency department of a tertiary referral center in central Israel. Patients admitted for facial nerve palsy (37 BP confirmed cases) were matched by age, sex, and date of admission with 72 controls admitted for other reasons and assessed against the odds of BNT162b2 vaccination. The odds of vaccination were not different between cases and controls. The odds ratio for vaccination was 0.84 (95% CI: 0.37–1.90) (Shemer et al., 2021). Shoaibi et al. (2023) conducted an SCCS study of BNT162b2 and mRNA-1273 among U.S. Medicare beneficiaries aged 65+ to evaluate association with BP after only a booster dose (Shoaibi et al., 2023). The study included 6.2 million individuals. Of 79 cases identified through electronic health records, chart reviews determined that 10 were confirmed or probable, for a positive predictive value of 12.66 percent. After adjusting for outcome misclassification, they found no significant association between BNT162b2 and BP (IRR 1.13, 95% CI: 0.77–1.65) or mRNA-1273 and BP (IRR 1.02, 95% CI: 0.70–1.50) (Shoaibi et al., 2023). In addition to these studies that evaluated individual vaccines, two studies evaluated the association of mRNA vaccines with risk of BP. Takeuchi et al. (2022) evaluated BP risk after any BNT162b2 and mRNA-1273 in administrative claims data using a cohort study design and an SCCS design. BP was defined by ICD codes from hospitalized claims data. The study Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 85 PREPUBLICATION COPY—Uncorrected Proofs included 136,644 people who received one dose, 127,268 who received two doses, and 183,990 unvaccinated. The vaccinees had two BP cases 21 days after dose 1 and one BP case after dose 2 compared with 18 cases among the unvaccinated. The adjusted IRR of BP was 1.14 (95% CI: 0.27–4.89) and 0.60 (95% CI 0.08–4.49) after dose 1 and dose 2, respectively, compared with unvaccinated. The results of the SCCS analysis indicated no increased risk of BP after dose 1 (IRR 1.03, 95% CI: 0.20–5.31) or dose 2 (IRR 0.47, 95% CI: 0.05–4.18) (Takeuchi et al., 2022). Klein et al. (2021) conducted a surveillance study within VSD. They compared incidence of BP 1–21 days after either dose 1 or 2 of an mRNA vaccine with that of concurrent comparators who, on the same calendar day, had received their most recent dose 22–42 days earlier. After 11.8 million doses, 535 BP cases were identified in the risk interval compared with 301 in the controlled interval. The adjusted IRR was 1.00 (95% CI: 0.86–1.17). In a supplemental analysis comparing vaccinated with unvaccinated people, they found no risk association with an mRNA vaccine (RR 1.06, 95% CI: 0.95–1.17) (Klein et al., 2021). From Evidence to Conclusions Among the 11 epidemiology studies reviewed, only one reported a significantly increased risk of BP after the first dose of BNT162b2 (Shibli et al., 2021). Its results are prone to confounding because it used historical BP rate as the comparator. Factors associated with that rate may be very different from those during the pandemic. Furthermore, comparing vaccinated with unvaccinated is problematic because, without randomization, it is practically impossible to balance their confounding factors. Although informative, this study weakly contributed to the final conclusion because of its limitations including using historical background rates as comparators; studies using concurrent comparators did not find an association between BP and mRNA vaccines. The main limitation is that most of the studies relied on ICD codes from electronic data without chart confirmation. Some cases of BP identified by the ICD codes might not be true or incident cases, which could have biased the measure of association. Studies may have missed cases because they were not based on active surveillance, and the majority of the cases included are likely more severe, as those with mild symptoms may not have sought medical attention during the pandemic. Furthermore, some studies may have incompletely measured or adjusted for some confounding. Conclusion 3-9: The evidence favors rejection of a causal relationship between the BNT162b2 vaccine and Bell’s Palsy. Conclusion 3-10: The evidence favors rejection of a causal relationship between the mRNA-1273 vaccine and Bell’s Palsy. Only two of the 11 studies evaluated the relationship between Ad26.COV2.S and BP; neither showed an increased risk. No studies evaluated the relationship between BP and NVX- CoV2373. Conclusion 3-11: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and Bell’s Palsy. Conclusion 3-12: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and Bell’s Palsy. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 86 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs TRANSVERSE MYELITIS BOX 3-4 Conclusions for Transverse Myelitis Conclusion 3-13: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and transverse myelitis. Conclusion 3-14: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and transverse myelitis. Conclusion 3-15: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and transverse myelitis. Conclusion 3-16: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and transverse myelitis. Background Spinal cord dysfunction of any cause is referred to as “myelopathy”; “myelitis” designates inflammation of the spinal cord. Acute TM refers to a group of acquired, acute-onset, focal inflammatory myelopathies. Consensus diagnostic criteria that rely on clinical and radiographic features have been published, and the diagnosis requires bilateral (although not necessarily symmetric) weakness and sensory deficits, with a clearly defined sensory level, evidence of inflammation by CSF or MRI gadolinium enhancement, and clinical progression to nadir between 4 hours and 21 days (Transverse Myelitis Consortium Working Group, 2002). This clinicoradiologic syndrome can be a manifestation of other inflammatory central nervous system disorders (disease-associated TM), including demyelinating disorders, such as neuromyelitis optica spectrum disorder, acute disseminated encephalomyelitis (ADEM), where up to 50 percent of patients have antibodies to myelin oligodendrocyte glycoprotein, and multiple sclerosis (MS) (Lopez Chiriboga, 2021). Spinal cord infections, paraneoplastic autoimmune syndromes, and systemic inflammatory disorders can also present as disease- associated TM (Flanagan et al., 2016; Jain et al., 2023). When the etiology is unknown, it is called “idiopathic TM.” Confusingly, noninflammatory causes of myelopathy, such as ischemic or hemorrhagic stroke, nutritional deficiencies, and neoplasms, can mimic this clinical and radiographic picture. In one study, 70 percent of patients referred to a tertiary care center with a diagnosis of idiopathic TM had a more specific disease-associated TM, such as myelin oligodendrocyte glycoprotein antibody–associated disease or MS, but a quarter of them did not have an inflammatory myelopathy at all (Zalewski et al., 2019). Idiopathic TM is therefore a diagnosis of exclusion (of known causes of disease-associated TM and noninflammatory myelopathies that can mimic TM). Another study, based on retrospective review of Veterans Health Administration electronic medical records, found that 57.6 percent of patients assigned an ICD code of TM lacked CSF testing, which is a core feature of current diagnostic criteria (Abbatemarco et al., 2021). As the aforementioned studies suggest, existing criteria lack specificity, which can affect the accuracy of epidemiological studies, especially those relying on ICD codes. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 87 PREPUBLICATION COPY—Uncorrected Proofs Idiopathic TM is rare, with a reported incidence of 1.34–4.6 per million per year, with bimodal peaks between ages 10–19 and 30–39 years and no sex predisposition (Bhat et al., 2010). It has been reported a few weeks after vaccination, although a large retrospective cohort study from VSD, a collaboration between the Centers for Disease Control and Prevention’s Immunization Safety Office and several integrated health care systems across the United States, did not find an increased risk in association with routine vaccines (Baxter et al., 2016). Mechanisms The pathophysiology of idiopathic TM is unknown, but postinfectious immune-mediated injury is the most widely accepted mechanism. This could be due to bystander activation, epitope spreading or polyclonal activation of previously dormant self-reactive lymphocytes (Chapter 2). Up to 40 percent of TM cases follow an infection, most commonly Coxsackie viruses and mycoplasma pneumoniae, and infectious agents have sometimes been isolated from the spinal fluid (Bhat et al., 2010; Krishnan et al., 2004). TM has also been reported after a variety of vaccines, including hepatitis B, rabies, and rubella (Agmon-Levin et al., 2009). The fact that TM has been associated with many different viruses and vaccines argues against molecular mimicry as a mechanism. In England in 1922–1923, over 200 cases of encephalomyelitis were reported after smallpox and rabies vaccination, and autopsy studies revealed inflammatory cells and demyelination in the spinal cord (Krishnan et al., 2004; Rivers, 1932). More recent pathological studies demonstrate focal infiltrates of monocytes and lymphocytes in the spinal cord and perivascular space, astroglial and microglial activation, and involvement of both white and gray matter (Krishnan et al., 2004). In the acute phase, heavy infiltration by CD4+ and CD8+ T cells and monocytes is found, whereas the subacute phase is characterized by macrophage infiltration and demyelination (Krishnan et al., 2004). Most patients with TM have CSF pleocytosis suggesting breakdown of the blood–brain barrier (Bhat et al., 2010; Krishnan et al., 2004). Patients with TM have been shown to have elevated levels of interleukin-6 (IL-6) in their CSF and, in acute TM, CSF IL-6 levels correlate with the ultimate level of clinical disability (Kaplin et al., 2005). In an animal model, IL-6 can be shown to mediate cord injury by inducing nitric oxide production, which is associated with oligodendrocyte injury, demyelination, and axonal injury (Kaplin et al., 2005). Epidemiological Evidence Clinical trial results submitted to FDA for Emergency Use Authorization and/or full approval do not indicate a signal regarding TM and any of the vaccines under study (FDA, 2021, 2023a, 2023b, 2023c). Table 3-5 presents five studies that contributed to the causality assessment. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 88 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TAB L E 3 -5 Ep i d e m i o l o g i c a l S t u d i e s i n t h e T r a n s v e r s e M y e l i t i s E v i d e n c e R e v i e w Au t h o r St u d y D e s i g n an d C o n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) Kl e i n e t a l . (2 0 2 1 ) Co h o r t / va c c i n a t e d co n c u r r e n t co m p a r a t o r s US VS D / E M R BN T 1 6 2 b 2 ≥1 6 ye a r s 6. 8 mi l l i o n do s e s BN T 1 6 2 b 2 an d mR N A -12 7 3 co m b i n e d : 2 BN T 1 6 2 b 2 an d mR N A -12 7 3 co m b i n e d : RR 1 . 4 5 (0 . 1 0 –47 . 7 3 ) mR N A -12 7 3 5. 1 mi l l i o n do s e s Li e t a l . ( 2 0 2 2 ) Co h o r t / se l f - co n t r o l l e d an d h i s t o r i c a l ba c k g r o u n d UK a n d Sp a i n Pr i m a r y c a r e da t a b a s e s li n k e d t o ho s p i t a l da t a / E M R BN T 1 6 2 b 2 (U K a n d S p a i n ) ≥1 8 ye a r s 3. 6 mi l l i o n va c c i n e e s UK : Do s e 1 : < 5 Do s e 2 : N o ca s e s Sp a i n : Do s e 1 : < 5 Do s e 2 : N o ca s e s SI R n o t ca l c u l a t e d mR N A -12 7 3 (S p a i n o n l y ) 24 4 , 9 1 3 va c c i n e e s Do s e 1 : No c a s e s Do s e 2 : < 5 Ad 2 6 . C O V 2 . S (S p a i n o n l y ) 12 0 , 7 3 1 va c c i n e e s Do s e 1 : N o ca s e s Pa t o n e e t a l . (2 0 2 1 ) Co h o r t / s e l f - co n t r o l l e d UK En g l i s h im m u n i z a t i o n re c o r d s / E M R BN T 1 6 2 b 2 ≥1 6 ye a r s 12 . 1 mi l l i o n va c c i n e e s 68 BN T 1 6 2 b 2 IR R 1 . 0 2 (0 . 7 5 –1. 4 0 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . NE U R O L O G I C CO N D I T I O N S 89 PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 3 -5 Co n t i n u e d Au t h o r St u d y D e s i g n an d C o n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) St u r k e n b o o m e t al . ( 2 0 2 2 ) Co h o r t / ba c k g r o u n d ra t e Eu r o p e a n co u n t r i e s Pr i m a r y a n d se c o n d a r y c a r e da t a b a s e s / E M R BN T 1 6 2 b 2 Va r i e d 6. 5 mi l l i o n do s e s 9 BN T 1 6 2 b 2 IR R 1 . 8 8 (0 . 3 7 –9. 6 0 ) mR N A -12 7 3 72 7 , 0 4 7 do s e s No c a s e s N/ A Ad 2 6 . C O V 2 . S 24 2 , 3 4 9 do s e s No c a s e s N/ A Wa l k e r e t a l . (2 0 2 2 ) Co h o r t / S e l f - co n t r o l l e d UK EM R BN T 1 6 2 b 2 ≥1 8 5. 7 mi l l i o n do s e s 10 9 IR R 1 . 4 9 (0 . 7 1 –3. 1 0 ) mR N A -12 7 3 25 5 , 4 4 6 do s e s No c a s e s N/ A NO T E S : B N T 1 6 2 b 2 r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y P f i z e r -Bi o N T e c h u n d e r t h e n a m e C o m i r n a t y ® . mR N A -12 7 3 r e f e r s t o t h e CO V I D -19 v a c c i n e m a n u f a c t u r e d b y M o d e r n a u n d e r t h e n a m e S p i k e v a x ® . A d 2 6 . C O V 2 . S r e f e r s t o t h e C O V I D -19 v a c c i n e m a n u f a c t u r e d b y Ja n s s e n . Th e p r i m a r y s e r i e s f o r A d 2 6 . C O V 2 . S i s o n e d o s e . N u m b e r o f e v e n t s r e f e r s t o e v e n t s i n v a c c i n e e s o n l y . CI : c o n f i d e n c e i n t e r v a l ; E M R : el e c t r o n i c m e d i c a l r e c o r d ; I R R : i n c i d e n c e r a t e r a t i o ; N / A : n o t a p p l i c a b l e ; R R : r a t e r a t i o . SO U R C E S : K l e i n e t a l . , 2 0 2 1 ; L i e t a l . , 2 0 2 2 b ; P a t o n e e t a l . , 2 0 2 1 ; S t u r k e n b o o m et a l . , 2 0 2 2 ; W a l k e r e t a l . , 2 0 2 2 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 90 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs Klein et al. (2021) conducted a surveillance study within VSD comparing TM incidence 1–21 days after either dose 1 or 2 of a mRNA vaccine with that of vaccinated concurrent comparators who, on the same calendar day, had received their most recent dose 22–42 days earlier. After 11.8 million doses (57 percent BNT162b2), two cases were identified in the risk interval compared with one in the controlled interval, with an adjusted rate ratio of 1.45 (95% CI: 0.10–47.73) and excess cases of 0.1 (95% CI: -1.6–0.2) risk interval per million doses (Klein et al., 2021). Li et al. (2022b) compared TM rates identified through medical records among vaccinees with historical background rates and conducted an SCCS analysis. They used data from CPRD AURUM and SIDIAP. The study included 3.6 million people who received BNT162b2, 244,913 who received mRNA-1273, 120,731 who received Ad26.CoV.2, and 14.3 million people from the general population. Of the BNT162b2 vaccinees, fewer than five cases occurred within 1–21 days after a first dose in CPRD AURUM, compared with 4.7 expected. SIDIAP had <5 cases after the first dose of BNT162b2, compared with 0.9 expected. For mRNA-1273, <5 cases were diagnosed after the second dose compared with 0.1 expected. No cases were observed with the second dose of BNT162b2, first dose of mRNA-1273, or Ad26.COV2.S. Walker et al. (2022) analyzed primary care data from more than 17 million patients in England linked to emergency care, hospital admission, and mortality records in OpenSAFELY. They used an SCCS analytical approach where the risk interval was 4–28 days after vaccination. Among 5.7 million recipients of BNT162b2, 109 TM cases were identified during the risk and controlled periods, and none were identified among 255,446 recipients of mRNA-1273. They found no significant association between the first dose of BNT162b2 and TM (IRR 1.62, 95% CI: 0.86–3.03). Few events were observed, so they were unable to precisely estimate the risk association. Adjusting for calendar time or history of COVID-19 infection did not significantly change the measure of association (IRR 1.49, 95% CI: 0.71–3.10) (Walker et al., 2022). Sturkenboom et al. (2022) conducted a cross-national multi-database retrospective dynamic cohort study using primary and/or secondary health care data from four European countries: Italy, the Netherlands, the United Kingdom, and Spain. They compared TM incidence in vaccine recipients with nonvaccinated persons in 2020 within 28 days after each dose. Of 25.7 million people, nine cases were identified after BNT162b2 (IRR 1.88, 95% CI: 0.37–9.6) and none after mRNA-1273 and Ad26.COV2.S (Sturkenboom et al., 2022). The results are consistent with an increased risk, but few events were observed, and the authors were unable to precisely estimate risk and results; this could also be consistent with no or decreased risk. Patone et al. (2021) investigated the association between BNT162b2 with potential neurological harms among 32.6 million vaccinees, 12.1 million of whom received BNT162b2 (Patone et al., 2021). An ICD-10 code for TM was included in the category “acute demyelinating events” (ADE), which contained ICD-10 codes for other demyelinating syndromes, such as ADEM. This retrospective self-controlled cohort study compared the incidence rate at several intervals (1–7, 8–14, 15–21, 22–28, and 1–28 days) after vaccination with that during periods outside of this interval. Sixty-eight events were observed after BNT162b2 during the risk period. They found no association for BNT162b2 at any interval, including in the 1–28 days period (IRR 1.02, 95% CI: 0.75–1.40). Few events were observed, so the authors were unable to precisely estimate the risk; the results would be consistent with no increased risk but also with slightly increased risk. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 91 PREPUBLICATION COPY—Uncorrected Proofs From Evidence to Conclusions The main limitation of the reviewed studies is their reliance on ICD codes from electronic data without chart confirmation. In addition, the studies used varying nomenclature when designating cases of vaccine-associated myelitis. Most had TM as a stand-alone adverse event, but one (Patone et al., 2021) included ICD codes for TM within the larger category “acute demyelinating events,” which also included ICD codes for other central nervous system inflammatory disorders. Three studies (Klein et al., 2021; Li et al., 2022; Patone et al., 2021) included a separate category “encephalitis/myelitis/encephalomyelitis,” and cases clinically and radiographically consistent with TM may have been classed within this category based on their ICD code, resulting in a lower number of total reported events. None of the five epidemiology studies suggested a causal association between TM and BNT162b2, and no evidence suggests a large association. However, the limited number of studies, along with the overall low number of events reported, raises the concern that a small association may have been missed, given that TM is a very rare disorder. Four studies included a few mRNA-1273 recipients, with no TM cases reported in two of the studies. Only one study included patients who received Ad26.COV2.S, with a comparatively low number of vaccinees and no cases reported (Li et al., 2022). Conclusion 3-13: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and transverse myelitis. Conclusion 3-14: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and transverse myelitis. Conclusion 3-15: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and transverse myelitis. Conclusion 3-16: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and transverse myelitis. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 92 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs CHRONIC HEADACHE BOX 3-5 Conclusions for Chronic Headache Conclusion 3-17: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and chronic headache. Conclusion 3-18: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and chronic headache. Conclusion 3-19: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and chronic headache. Conclusion 3-20: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and chronic headache. Background Headache is a frequently reported symptom of systemic illness, cerebrovascular disorders, intracranial disease, or craniocervical trauma. It is also reported commonly and can be a symptom of substance withdrawal. When a headache results from a separate medical condition, it is called a “secondary headache.” Most headaches, however, occur as the principal manifestation of a primary headache disorder; these are characterized by recurrent headaches of varying characteristics, frequency, and accompanying symptoms and signs. Although the frequency and severity of individual headache episodes vary over the lifetime, primary headache disorders are usually considered lifelong conditions. They have no biological markers, and their diagnosis is made with reasonable precision based on consensus diagnostic criteria set forth in the International Classification of Headache Disorders (ICHD-3), which was last revised in 2018 (International Headache Society, 2018). Ancillary studies, mostly brain and vascular imaging and occasionally lumbar puncture, are used to rule out various forms of secondary headaches. Tension-type headache (TTH) and migraine are by far the more common primary headache disorders, with an estimated lifetime prevalence in the general population of 46 and 14 percent, respectively (Stovner et al., 2007). Geographic variations exist, but it is unclear whether these are driven by genetic differences or methodological differences between studies. Other primary headache disorders, such as cluster headache, are much rarer, with a lifetime prevalence of 0.06–0.3 percent (Jensen and Stovner, 2008). The frequency, duration, and severity of headache varies significantly even within the same primary headache disorder: from infrequent, short, and mild to continuous and/or disabling. Migraine is more common in women compared to men, with a ratio of 2:1 to 3:1 (Jensen and Stovner, 2008). The female:male ratio for TTH is 5:4 (Jensen and Stovner, 2008). The prevalence of migraine peaks between the second and third decades of life but can affect people of all ages, including children. Data regarding age dependence in TTH are more limited, but prevalence peaks around the fourth decade of life. Cluster headache has a male:female ratio of 4.3:1 (Fischera et al., 2008), with prevalence peaking between the second and fourth decades of life. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 93 PREPUBLICATION COPY—Uncorrected Proofs No single consensus diagnostic criteria exist for chronic headache. Rather, ICHD-3 provides diagnostic criteria for chronic forms of individual headache subtypes based on frequency and duration. These include chronic migraine headache, chronic TTH, chronic cluster headache, hemicrania continua, new daily persistent headache, and medication overuse headache, which is a form of secondary headache (International Headache Society, 2018). In most, but not all, chronicity is based on a frequency of more than 15 headache days per month for longer than 3 months. Although ICHD-3 criteria for secondary headache does not specify measures of chronicity, they do specify that when a pre-existing primary headache becomes chronic shortly after a known causative disorder, both chronic primary headache and secondary headache diagnoses should be given (International Headache Society, 2018). Data on chronic headaches is relatively scarce, but prevalence as a group is estimated as 3–4 percent in the general population (Jensen and Stovner, 2008). Systemic infection, including with COVID-19, can be associated with headache (Togha et al., 2022), and “headache attributed to systemic infection” is included as a subtype of secondary headache in ICHD-3. Headache was also a frequently reported symptom in the clinical trials for the various COVID-19 vaccines (Baden et al., 2021; Heath et al., 2021; Polack et al., 2020; Sadoff et al., 2021). Most of these headaches occurred within 24 hours of vaccination and were frequently accompanied by systemic symptoms, such as fatigue, fever, chills, and myalgia (Göbel et al., 2021a, 2021b). In most, headaches lasted less than 72 hours, with only a small minority reporting more than 3 days. Pre-existing migraine was associated with more severe and long-lasting headaches in some but not all studies (Silvestro et al., 2021) and may predispose someone to postvaccine headache (Sekiguchi et al., 2022). Although ICHD-3 does include “headache attributed to use or exposure to a substance” as a subtype of secondary headache, vaccines are not listed within the known causes (International Headache Society, 2018). Evidence suggests that headache may be common with other vaccines as well, and some have proposed that postvaccination headache should be included in the next iteration of the ICHD (Garces et al., 2022). Headache is also one of the main symptoms of cerebral venous sinus thrombosis (CVST), a manifestation of vaccine-induced immune thrombotic thrombocytopenia (VITT). VITT has been reported in association with the AV COVID-19 vaccines and is discussed elsewhere in this report (See et al., 2021). Unlike the more common postvaccination headache, which occurs shortly after vaccination, the headache secondary to VITT-associated CVST is approximately a week after vaccination (García-Azorín et al., 2021). Mechanism The pathophysiology of primary headache disorders remains ill-defined and is different for individual disorders. Postvaccination headache is not included as a type of secondary headache in ICHD-3; however, it may bear some resemblance to “headache attributed to systemic infection,” which is included. The more widely accepted hypothesis is that postvaccination headache is secondary to downstream effects stemming from the immune response to the vaccine (Garces et al., 2022). Vaccines, including COVID-19 vaccines, are associated with the release of inflammatory mediators, such as prostaglandin E, and proinflammatory cytokines. It is conjectured that these are responsible for the headache and frequently associated systemic symptoms. Some have proposed that inflammatory mediators may modulate the release of calcitonin gene–related peptide (CGRP), which plays an important role in migraine via activation of the trigeminovascular system. Similarly, substance P, a Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 94 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs nociceptive neuropeptide released by trigeminal sensory fibers and implicated in migraine, is also produced by mast cells, suggesting a link between immune activation and migraines (Suvas, 2017). Data supporting this hypothesis are limited. One study found increased levels of inflammatory and nociceptive molecules in COVID-19 hospitalized patients with headache compared to those without; CGRP levels, however, did not differ significantly between the two groups (Bolay et al., 2021). Finally, some have hypothesized direct modulation of the trigeminal nerve when the spike protein, which is either synthetized intracellularly or introduced directly after vaccination, binds the ACE2 receptor. However, it remains unclear whether ACE2 is expressed in the relevant neural structures (Caronna et al., 2023), and some studies suggest that headache is more common after the second dose (Ceccardi et al., 2022), which appears counterintuitive given the probable presence of neutralizing antibodies against the spike protein Epidemiological Evidence Chronic headache is not a single diagnostic entity with widely accepted diagnostic criteria. The committee relied on ICHD-3, which provides diagnostic criteria for the subtypes. Although a self-limited headache was a commonly reported symptom after BNT162b2, mRNA- 1273, Ad26.COV2.S, and NVX-CoV2373, none of the studies reviewed included a stand-alone category for chronic headache, nor did they include chronic headache subtypes as defined in ICHD-3. Clinical trial results submitted to FDA for Emergency Use Authorization and/or full approval do not indicate a signal regarding chronic headache and any of the vaccines under study (FDA, 2021, 2023a, 2023b, 2023c). None were included in the final report for analysis. From Evidence to Conclusions The epidemiological and mechanistic literature are absent regarding the relationship between COVID-19 vaccines and chronic headache. Conclusion 3-17: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and chronic headache. Conclusion 3-18: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and chronic headache. Conclusion 3-19: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and chronic headache. Conclusion 3-20: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and chronic headache. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 95 PREPUBLICATION COPY—Uncorrected Proofs POSTURAL ORTHOSTATIC TACHYCARDIA SYNDROME BOX 3-6 Conclusions for Postural Orthostatic Tachycardia Syndrome Conclusion 3-21: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and postural orthostatic tachycardia syndrome. Conclusion 3-22: The evidence is inadequate to accept or reject a causal relationship between the mRNA -1273 vaccine and postural orthostatic tachycardia syndrome. Conclusion 3-23: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and postural orthostatic tachycardia syndrome. Conclusion 3-24: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and postural orthostatic tachycardia syndrome. Background POTS is marked by symptoms of orthostatic intolerance despite relative preservation of autonomic reflexes. The hallmark is an exaggerated increase in heart rate in response to standing or tilt without a drop in blood pressure as seen in classic autonomic failure (Cutsforth-Gregory, 2020). POTS is defined as a sustained heart rate increase of 30 beats per minute (bpm) or increase to 120 bpm within the first 10 minutes of orthostasis, along with symptoms of orthostatic intolerance, including dizziness, palpitations, weakness, and tremulousness. For children and adolescents (12–19 years), the required increment is 40 bpm (Vernino et al., 2021). POTS predominantly affects a younger and primarily female (at a ratio of 4:1) demographic, with the typical age range of onset being 12–50 (Vernino et al., 2021). Epidemiologically, it is a relatively common condition in developed countries, with prevalence estimates of 0.2–1.0 percent of the U.S. population, which represents 1–3 million people (Cutsforth-Gregory, 2020; Vernino et al., 2021). Orthostatic symptoms are probably driven by both cerebral hypoperfusion (dizziness, lightheadedness, vision, and hearing changes) and sympathoexcitation (palpitations, chest pain, difficulty breathing, tremulousness, sweating, and coldness of the extremities) (Cutsforth- Gregory, 2020). Particularly tachycardia, can be triggered either directly by influencing the sinus rate control system via adrenergic and muscarinic receptors or indirectly as a compensatory response to peripheral vasodilation. This indirect response may involve adrenergic, angiotensin, and other potential vasoactive receptors (Figure 3-2). POTS patients, however, frequently experience other symptoms as well, including sleep disturbances, headache, fatigue, cognitive impairment, gastrointestinal complaints, urinary frequency, and exercise intolerance (Vernino et al., 2021). The sheer variety and nonspecificity of these symptoms make it difficult to attribute all of them to a single clinical entity sharing the same underlying mechanism. A variety of comorbid conditions are associated with POTS, including migraine, somatic hypervigilance, irritable bowel syndrome, hypermobile Ehlers-Danlos syndrome (EDS), mast cell activation syndrome, systemic autoimmune disease, small-fiber neuropathy, and fibromyalgia and chronic fatigue syndrome (Gradin et al., 1987; Low et al., 2009; Shibao et al., 2005). It is unclear Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 96 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs whether the presence of these diagnoses defines unique pathophysiological subsets (Vernino et al., 2021). In either case, the diagnostic criteria emphasize symptoms and heart rate increment in response to an orthostatic challenge as the core feature, which is appropriate, as an excessive heart rate is the most consistent and reproducible of various indexes of orthostatic intolerance (Vernino et al., 2021). Symptoms alone in the absence of orthostatic tachycardia cannot be used to make the diagnosis and the syndrome must be present for at least 3 months (Vernino et al., 2021). The diagnostic approach begins with a comprehensive clinical assessment focused on orthostatic intolerance symptoms. Excessive increase in heart rate without orthostatic hypotension within 3–10 minutes from standing should be confirmed at bedside or with a tilt- table test (Freeman et al., 2011; Vernino et al., 2021). Laboratory tests play an important role in excluding other conditions that might mimic POTS symptoms. Further autonomic testing and/or skin biopsy may be warranted to explore the full spectrum of autonomic dysfunction and assess for underlying small-fiber neuropathy (Vernino et al., 2021). A 12-lead electrocardiography should be performed in all patients, but expanded cardiac evaluation, may be indicated in some (Cutsforth-Gregory, 2020). Between 20 and 50 percent of patients report a viral illness before the onset of symptoms. In these cases, POTS symptoms appear to arise abruptly weeks after the acute illness, but in others, the symptoms appear slowly (Thieben et al., 2007). Other triggers include surgery, and head trauma, although these are less well established (Olshansky et al., 2020). Patients have developed POTS symptoms at the time of or within 6 weeks of acute SARS-CoV-2 infection (Goodman et al., 2021), but latency can be longer, and POTS is considered a phenotype of postacute or “long” COVID-19 (Fedorowski and Sutton, 2023). POTS has also been reported in association with the COVID-19 vaccine (Kwan et al., 2022). Mechanisms The pathophysiology of POTS remains ill defined, and it is unlikely that it is a single disorder. Rather, it is probably a heterogeneous syndrome that can arise in various clinical scenarios resulting from distinct but overlapping pathophysiologic mechanisms (Benarroch, 2012). Several mechanisms have been proposed and account for some of its phenotypic variability. These include catecholamine excess (hyperadrenergic POTS), sympathetic denervation leading to impaired vasoconstriction of the lower limbs (neuropathic POTS), volume dysregulation, and deconditioning (Vernino et al., 2021). The clinical picture with hyperadrenergic POTS is dominated by palpitations, sweating, tremulousness, and orthostatic hypertension. Some of these patients have high plasma norepinephrine concentrations during orthostasis (Fedorowski and Sutton, 2023), although in others, the hyperadrenergic state may be secondary to medications, such a tricyclic antidepressants or methylphenidate (Cheshire, 2016). Neuropathic POTS may be secondary to a length-dependent autonomic neuropathy leading to impaired vasomotor tone in the lower limbs. Autonomic testing in some patients demonstrates loss of sweating in the feet and reduced increment of norepinephrine in the lower limbs when standing, which is consistent with a length- dependent autonomic neuropathy. The etiology of this autonomic neuropathy is not usually evident, although several lines of evidence suggest a potential immune-mediated mechanism in some cases. Reports of an earlier viral illness in up to one-half of patients suggests a postinfectious autoimmune process (Sandroni et al., 1999; Vernino et al., 2021). In addition, several small studies have demonstrated higher levels of functionally active antibodies to G- Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 97 PREPUBLICATION COPY—Uncorrected Proofs protein-coupled adrenergic receptors α1 and α2 in individuals with POTS than in healthy controls (Fedorowski and Sutton, 2023; Kharraziha et al., 2020; Li et al., 2014; Vernino et al., 2021). These findings, plus reports of the successful treatment of POTS with intravenous immunoglobulin (Rodriguez et al., 2021; Weinstock et al., 2018), suggest an autoimmune etiology, at least in a subset of patients. However, a recent randomized control trial of IVIg in POTS found no difference in symptom response compared to albumin infusion (Vernino, 2023). Most patients have some degree of hypovolemia. Studies have demonstrated that many of them have low levels of plasma-renin activity and aldosterone compared with controls (Raj et al., 2005), and some have reduced ACE2 activity (Stewart et al., 2009). The excessive venous pooling that occurs with vasomotor impairment in neurogenic POTS can lead to reduced cardiac preload and capillary leakage upon standing with associated net loss of plasma volume (Cutsforth-Gregory, 2020). In those with poor oral intake or excess fluid loss, such as in irritable bowel syndrome, managing the primary disorder will improve orthostatic intolerance. Finally, physical deconditioning can lead to orthostatic intolerance. Many patients show evidence of deconditioning: reduced stroke volume and left ventricular mass and persistent tachycardia and reduced peak oxygen when standing or exercising (Fu et al., 2010; Masuki et al., 2007). POTS has been reported in association with SARS-CoV-2 infection (Kwan et al., 2022; Miglis et al., 2020), including in patients with post-acute COVID-19 (Fedorowski and Sutton, 2023). However, caution is needed when assessing the literature because, although orthostatic intolerance is commonly reported in patients with post-acute COVID-19, many may not meet diagnostic criteria for POTS. In one study of patients with de novo orthostatic intolerance after COVID-19, only 22 percent fulfilled criteria for POTS (Shouman et al., 2021); the symptoms may be driven by deconditioning in some of these patients. In addition to POTS, small-fiber neuropathy, which can cause autonomic dysfunction and a POTS phenotype, has been described after COVID-19, including in post-acute COVID-19 (Abrams et al., 2022; Oaklander et al., 2022). POTS has also been reported after COVID-19 vaccination (Kwan et al., 2022). Many of these reports postulate an immune-mediated mechanism, but definitive evidence is lacking. One study demonstrated elevated inflammatory cytokines and markers of autoimmunity in patients presenting with POTS after COVID-19, although this study did not include relevant controls. One in silico study identified a variety of SARS-CoV-2 amino acid sequences, including in the spike protein, that are also present in vagal nuclei and ganglia (Marino Gammazza et al., 2020). This raises the theoretical possibility that molecular mimicry could induce cross-reactive immune responses resulting in low vagal tone after infection or vaccination. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 98 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs FIGURE 3-2 Postulated mechanisms of orthostatic intolerance and tachycardia in POTS. SOURCE: Fedorowski et al., 2017. Epidemiological Evidence Clinical trial results submitted to FDA for Emergency Use Authorization and/or full approval do not indicate a signal regarding POTS and any of the vaccines under study (FDA, 2021, 2023a, 2023b, 2023c). Table 3-6 summarizes one study that contributed to the causality assessment. TABLE 3-6 Epidemiological Study in the Postural Orthostatic Tachycardia Syndrome Evidence Review Author Study Design and Control Group Location Data Source Vaccine(s) Age Range Sample Size Number of Events Results (95% CI) Kwan et al. (2022) Cohort/self- controlled US EMR BNT162b2 ≥12 years 284,592 patients (62.2%) 763 events per 100,000 POTS cases during OR 1.52 (1.36– 1.71) mRNA- 1273 31% Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. NEUROLOGIC CONDITIONS 99 PREPUBLICATION COPY—Uncorrected Proofs Ad26.COV 2.S 6.9% exposure period compared to 501 per 100,000 pre- exposure NVX- CoV2373 <1% other* NOTES: BNT162b2 refers to the COVID-19 vaccine manufactured by Pfizer-BioNTech under the name Comirnaty®. mRNA-1273 refers to the COVID-19 vaccine manufactured by Moderna under the name Spikevax®. Ad26.COV2.S refers to the COVID-19 vaccine manufactured by Janssen. NVX-CoV2373 refers to the COVID-19 vaccine manufactured by Novavax. *<0.1% of other vaccines includes ChAdOx1-S, NVX-CoV2373, and CoronaVac. Number of events refers to events in vaccinees only. CI: confidence interval; EMR: electronic medical record; OR: odds ratio. SOURCE: Kwan et al., 2022. Kwan et al. (2022) derived cohorts from the diverse patient population of the Cedars- Sinai Health System in Los Angeles County, California. The authors identified patients who had at least one COVID-19 vaccination between 2020 and 2022 and excluded those with a documented COVID-19 infection 90 days before or after vaccination (n = 5,070). The final sample was 284,592 patients (age 52 ± 20 years; 57 percent female; 63 percent White, 10 percent Asian, 8.9 percent African American, and 12 percent Hispanic). Among the sample, 62 percent received BNT162b2, 31 percent mRNA-1273, 6.9 percent Ad26.COV2.S, and less than 0.1 percent other vaccines. POTS was identified using diagnosis codes (ICD-9 I49.8; ICD-10 G90.9) and modeled as both a single diagnosis and a combination of POTS-associated diagnoses (POTS diagnosis codes, Fatigue, Dysautonomia, EDS, and mast cell disorders). Only outpatient encounters were used. From the 90-day prevaccination to 90-day postvaccination periods, the incidence of new diagnoses of POTS increased from 176 per 100,000 to 268 per 100,000 vaccinees (the authors did not report incidence per 100,000 for the combined diagnoses). Relative to the prevaccination period, the odds of a new diagnosis of POTS and POTS-associated diagnoses increased 52 percent, OR 1.52 (95% CI: 1.36–1.71) and 33 percent, OR 1.33 (95% CI: 1.25–1.41) in the postvaccination period, respectively. Limitations exist from unmeasured confounding, lack of inclusion of COVID-19 infection, and open nature of the dataset, as patients could have had encounters in other health systems as well. In addition, the measure of effect was calculated for all vaccines combined, and conclusions cannot be drawn regarding a potential association between POTS and individual vaccines or platforms. The committee also reviewed a case series (Eldokla and Numan, 2022) of five patients who developed de novo POTS within 21 days of an mRNA vaccine (four BNT162b, one mRNA- 1273). All five underwent detailed autonomic testing and met diagnostic criteria for POTS. Two had elevated proinflammatory cytokines, and two had mildly elevated autoantibodies (thyroid peroxidase antibodies and antinuclear antibodies), without other signs or symptoms of systemic autoimmune disease. One had a low titer of acetylcholine receptor ganglionic antibodies; at higher titers, this has been associated with autoimmune autonomic failure. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 100 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs From Evidence to Conclusions The totality of the evidence included one epidemiological study with methodological limitations and one case series with adequate case identification but no comparator group. No definitive mechanism was identified in the literature. Conclusion 3-21: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and postural orthostatic tachycardia syndrome. Conclusion 3-22: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and postural orthostatic tachycardia syndrome. Conclusion 3-23: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and postural orthostatic tachycardia syndrome. 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PREPUBLICATION COPY—Uncorrected Proofs 4 Sensorineural Hearing Loss, Tinnitus, and COVID-19 Vaccines This chapter describes the potential relationship between COVID-19 vaccines and sudden sensorineural hearing loss (SSNHL) and tinnitus (see Boxes 4-1 and 4-2 for all conclusions in this chapter). SENSORINEURAL HEARING LOSS BOX 4-1 Conclusions for Sensorineural Hearing Loss Conclusion 4-1: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and sensorineural hearing loss. Conclusion 4-2: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and sensorineural hearing loss. Conclusion 4-3: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and sensorineural hearing loss. Conclusion 4-4: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and sensorineural hearing loss. Background The whole auditory system is how humans’ access and make sense of environmental sounds. It is a multistage system characterized by encoding of environmental auditory stimuli by peripheral structures and decoding of the stimuli by central structures in the brainstem and cerebral cortex (Pickles, 2013). Peripherally, auditory energy is funneled into the pinna toward the tympanic membrane (eardrum), where it is converted to mechanical energy and moves along the ossicles in the middle ear to the cochlea, which contains the organ of Corti, which acts to encode auditory signals as neuroelectric signals (e.g., action potentials) that are transmitted to the temporal lobe via the eighth nerve and brainstem for decoding and processing (Pickles, 2013). Damage can occur at any step in this process, resulting in different types of hearing loss. Conductive hearing loss is characterized by an inability for the outer and middle ear to transmit signals to the inner ear (e.g., rupture in the tympanic membrane, fluid in the middle ear) and is Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 114 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs often transient (e.g., fluid drains from the ear) or can be addressed via medical or surgical interventions (Lee, 2013; Pickles, 2013). Sensorineural hearing loss is distinguished by disruption in encoding auditory information in the cochlea or along the eighth nerve and is usually permanent. Central hearing loss or auditory processing disorders (Martin and Jerger, 2005; Task Force on Central Auditory Processing Consensus Development, 1996), although more poorly understood and considered rare, especially among adults, occur when sound is encoded normally in the peripheral ear (e.g., no sign of sensorineural or conductive loss but deficits in the neural processing of auditory information mean that individuals struggle with understanding it despite functioning peripheral hearing (Katz, 2015). Audiologists and otolaryngologists diagnose hearing loss using a comprehensive assessment battery, including various measures assessing different processes of the auditory system (Katz, 2015). The criterion standard for peripheral hearing is pure-tone audiometry, which identifies the softest volume at which tones at different frequencies can be detected. A combination of methods of presenting the tone via air conduction (e.g., traditional headphones that stimulate the entire outer, middle, and inner ears) and bone conduction (e.g., oscillator that directly stimulates the cochlea) distinguish different types of hearing loss (Katz, 2015). Self-reported hearing has relatively poor agreement with the criterion standard, with sensitivity and specificity reported as 41–65 percent and 81–88 percent among U.S. adults over 20 years old, respectively (Agrawal et al., 2008). Moreover, accuracy and the direction of misclassification (e.g., directional difference between self-report and criterion measured degree of hearing loss) differs by key demographic variables, including age, race, and sex; older White men are more likely to underestimate their level of hearing loss relative to younger Black women (Kamil et al., 2015). The relatively poor accuracy of self-reported hearing can be attributed to the insidious onset of age-related hearing loss masking the change, perceived normalcy for a given age group, stigma, or projection (believing that others are mumbling or speaking poorly). Moreover, understanding speech requires both an auditory (e.g., accessing sound) and cognitive (e.g., making sense of the information) component, and listening with hearing loss can contribute to fatigue from cognitive load placed on the brain when decoding poor peripheral signals (Hornsby et al., 2016; Wingfield et al., 2005). Some may misattribute hearing loss to cognitive processes and vice versa when considering their own hearing levels. Although the procedures are standardized, the actual clinical cut points vary by professional organizations and are at the discretion of the provider. Population estimates vary by the definition and whether hearing loss estimates are limited to bilateral or unilateral (estimates increase when including unilateral loss) (Lin et al., 2011). Using the commonly cited World Health Organization (WHO) cutoffs from before 2021, estimates suggest that 23 percent of U.S. individuals over age 12 have bilateral hearing loss and that prevalence increases with age, from less than 1 percent at 20–29 years to more than 80 percent over 80 years (Goman and Lin, 2016). WHO suggests approximately 20 percent of the global population has hearing loss (WHO). Among the types of hearing loss, specific reliable national estimates are not reported. Permanent conductive hearing loss is relatively rare (Cruickshanks et al., 1998), and sensorineural hearing loss is the overwhelmingly most common permanent form, with the majority of cases being attributed to age (Reed et al., 2023; Yamasoba et al., 2013). However, estimates vary by definition of hearing loss and global region and are limited due to the often-transient nature of conductive hearing loss, relatively low uptake of hearing assessment within health systems, and lack of feasibility for comprehensive hearing assessment in epidemiological studies (Chadha et al., 2021; Katz, 2015; Powell et al., 2021). Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. HEARING LOSS AND TINNITUS 115 PREPUBLICATION COPY: Uncorrected Proofs Known individual risk factors for sensorineural hearing loss include congenital and progressive genetic conditions, excessive noise, certain medications and chemicals, health behaviors (e.g., smoking), chronic cardiovascular conditions, viral infections, and age-related cellular degeneration (Agrawal et al., 2008; Eggermont, 2017; Van Eyken et al., 2007). The majority of adult hearing loss is often labeled as “age-related” and attributed to a combination of exposures that insidiously degrades hearing acuity such that changes are so subtle they often go unnoticed until they are more pronounced (Lin et al., 2011; Yamasoba et al., 2013). SSNHL is characterized as an acute change (e.g., within a 72-hour period). The specific mechanisms are poorly characterized as several risk factors and potential causes have been reported including infection, trauma, autoimmune disease, certain medications (e.g., aminoglycosides), and certain disorders of the inner ear (e.g., Meniere’s) (Kuhn et al., 2011; Schreiber et al., 2010; Stachler et al., 2012). It is relatively rare (approximately 5–20 of 100,000 people yearly), but estimates are mostly reliant on high-income countries (Stachler et al., 2012). Estimates suggest that approximately 40–60 percent of cases will recover to normal levels in a few weeks of follow-up (Kuhn et al., 2011; Mattox and Simmons, 1977; Wilson et al., 1980). However, the incidence and recovery rate are not well documented in low- and middle-income countries. Moreover, there is variation in the literature of the different definitions for risk- windows and specific change in audiometric thresholds. Mechanisms The mechanistic evidence for a biologically plausible association between hearing loss and COVID-19 vaccination is limited; a paucity of work offers direct evidence. Similarly, there is little mechanistic evidence whether COVID-19 infection causes hearing loss. Much of the relevant literature is theoretical or postulated based on adjacent research. Moreover, no literature offers substantive discussion of the potential for increased risk of an association by comorbid conditions, genetic predisposition, concurrent pharmacologic agent, or environmental exposures. The initial consideration is the possible direct viral involvement of the inner ear or the vestibulocochlear nerve (Kaliyappan et al., 2022). The inflammatory response, possibly cochleitis or neuritis, could be an effect of the immune activation by the vaccine. The hyperproduction of proinflammatory cytokines in response to the vaccine could inadvertently affect the audio vestibular system, leading to symptoms such as vertigo, tinnitus, and hearing loss. Such a hyperinflammatory state is known to cause tissue damage and could be particularly detrimental to the sensitive structures of the ear (Kamogashira et al., 2022). Specifically, the response to BNT162b2 1 provides a hypothetical framework. Studies demonstrate that this vaccine elicits a strong immune response, characterized by high levels of neutralizing antibodies and robust T cell responses, including antigen-specific CD8+ and Th1-type CD4+ T cells (Sadarangani et al., 2021). Although this is crucial for protective immunity, it also raises the potential for unintended auditory effects. The inflammatory environment can indirectly inflict damage on the intricate anatomy of the ear, affecting or occluding small areas within it. The vigorous immune response, especially the aspects involving cell-mediated immunity and cytokine production, could inadvertently affect the ear through either direct inflammatory damage or secondary effects, such as vascular complications. 1 The COVID-19 vaccine manufactured by Pfizer-BioNTech under the name Comirnaty®. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 116 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs Others have postulated about molecular mimicry and immunological considerations, such as an autoimmune-like response, where antibodies or T cells, activated by the vaccine, might erroneously recognize inner ear antigens as viral epitopes and trigger an immune attack (Ahmed et al., 2022). Given the specificity and sensitivity of the immune response, particularly the adaptive immunity involving antigen-specific T cell and B cell responses, this cross-reactivity could be a plausible mechanism for vaccine-induced auditory damage. Last, the unique anatomical and physiological characteristics of the cochlea and semicircular canals, notably their isolated blood supply, make them particularly vulnerable to ischemic events (Tabuchi et al., 2010). Vaccine-induced alterations in the cardiovascular system, either directly or through an immune-mediated pathway, could lead to thrombosis or hypoxia in these areas, resulting in auditory dysfunction. Epidemiological Evidence Clinical trial results submitted to FDA for Emergency Use Authorization and/or full approval do not indicate a signal regarding sensorineural hearing loss and any of the vaccines under study (FDA, 2021, 2023a, 2023b, 2023c). Table 4-1 presents five studies that contributed to the causality assessment. Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . HE A R I N G L O S S A N D T I N N I T U S 11 7 PR E P U B L I C A T I O N C O P Y : Un c o r r e c t e d P r o o f s TA B L E 4 -1 Ep i d e m i o l o g i c a l S t u d i e s i n t h e Se n s o r i n e u r a l H e a r i n g L o s s Ev i d e n c e R e v i e w Au t h o r St u d y De s i g n and Co n t r o l Gr o u p Lo c a t i o n Da t a So u r c e Va c c i n e (S ) Ag e Ra n g e N Nu m b e r of Ev e n t s Re s u l t s (9 5 % CI ) Fo r m e i s t e r et a l . (2 0 2 2 ) Su r v e i l l a n c e ; si n g l e a r m US A VA E R S BN T 1 6 2 b 2 15 – 9 3 ye a r s 18 5 . 4 mi l l i o n do s e s 30 5 Al l v a c c i n e s : IR 0 . 6 ( p r o b a b l e ; mi n i m u m e s t i m a t e ) an d 2 8 . 0 ( m a x i m u m es t i m a t e ) c a s e s o f SS N H L p e r 1 0 0 , 0 0 0 pe o p l e p e r ye a r mR N A -1 2 7 3 22 2 Ad 2 6 . C O V 2 . S 28 Fr o n t e r a e t al . ( 2 0 2 2 ) Su r v e i l l a n c e ; si n g l e a r m US A VA E R S BN T 1 6 2 b 2 ≥12 ye a r s 16 7 . 0 mi l l i o n do s e s No t re p o r t e d 3. 2 0 p e r 1 mi l l i o n do s e s mR N A -1 2 7 3 12 8 . 1 mi l l i o n d o s e s 3. 0 8 p e r 1 mi l l i o n do s e s Ad 2 6 . C O V 2 . S 11 . 6 mi l l i o n do s e s 6. 2 9 p e r 1 mi l l i o n do s e s Le o n g e t al . ( 2 0 2 3 ) Cl i n i c a l co n v e n i e n c e sa m p l e ; si n g l e a r m NY , N Y , US A Ot o l o g y cl i n i c a t a n ac a d e m i c ce n t e r BN T 1 6 2 b 2 16 – 1 0 1 ye a r s 24 4 v a c c i n e e s 10 1. 7 % o f a l l va c c i n a t e d in d i v i d u a l s h a d ad j u d i c a t e d n e w he a r i n g l o s s mR N A -1 2 7 3 12 3 v a c c i n e e s 9 Ad 2 6 . C O V 2 . S 16 v a c c i n e e s 1 Ni e m i n e n et a l . (2 0 2 3 ) Po p u l a t i o n ba s e d Co h o r t ; p r e an d p o s t Fi n l a n d Fi n n i s h po p u l a t i o n in f o r m a t i o n sy s t e m BN T 1 6 2 b 2 0 t o ≥80 ye a r s 5. 5 m i l l i o n in d i v i d u a l s ( t o t a l co h o r t ) Do s e 1 : 1 1 1 Do s e 2 : 1 0 4 IR R 0 . 8 ( 0 . 6 – 1 . 0 ) IR R 0 . 8 ( 0 . 6 – 1 . 2 ) mR N A -1 2 7 3 Do s e 1 : 1 5 Do s e 2 : 2 0 IR R 0 . 8 ( 0 . 5 – 1 . 4 ) IR R 1. 2 ( 0 . 7 – 1 . 9 ) Ev i d e n c e R e v i e w o f t h e A d v e r s e E f f e c t s o f C O V I D - 1 9 V a c c i n a t i o n a n d I n t r a m u s c u l a r V a c c i n e A d m i n i s t r a t i o n Co p y r i g h t N a t i o n a l A c a d e m y o f S c i e n c e s . A l l r i g h t s r e s e r v e d . 11 8 VA C C I N E E V I D E N C E R E V I E W PR E P U B L I C A T I O N C O P Y —Un c o r r e c t e d P r o o f s TA B L E 4 -1 C o n t i n u e d Au t h o r St u d y De s i g n a n d Co n t r o l Gr o u p Lo c a t i o n Da t a S o u r c e Va c c i n e ( s ) Ag e Ra n g e N Nu m b e r o f Ev e n t s Re s u l t s (9 5 % C I ) Ya n i r e t a l . (2 0 2 2 ) Po p u l a t i o n ba s e d Co h o r t ; p r e an d p o s t Is r a e l Cl a l i t H e a l t h Se r v i c e s BN T 1 6 2 b 2 16 t o ≥65 ye a r s Do s e 1 : 2. 6 mi l l i o n va c c i n e e s 91 SI R 1 . 3 5 ( 1 . 0 9 – 1 . 6 5 ) Do s e 2 : 2. 4 mi l l i o n va c c i n e e s 79 SI R 1 . 2 3 ( 0 . 9 8 – 1 . 5 3 ) NO T E S : B N T 1 6 2 b 2 r e f e r s t o t h e CO V I D -19 v a c c i n e m a n u f a c t u r e d b y P f i z e r -B i o N T e c h u n d e r t h e n a m e C o m i r n a t y ® . m R N A - 1 2 7 3 r e f e r s t o t h e CO V I D -1 9 v a c c i n e m a n u f a c t u r e d b y M o d e r n a u n d e r t h e n a m e S p i k e v a x ® . A d 2 6 . C O V 2 . S r e f e r s t o t h e C O V I D - 1 9 v a c c i n e m a n u f a c t u r e d b y Ja n s s e n . T h e t h r e e a p p r o v e d CO V I D -1 9 v a c c i n e s i n t h e U K a r e B N T 1 6 2 b 2 , m R N A - 1 2 7 3 , a n d C h A d O x 1 - S. N u m b e r o f e v e n t s r e f e r s t o e v e n t s i n va c c i n e e s o n l y . C I : c o n f i d e n c e i n t e r v a l ; I Q R : i n t e r q u a r t i l e r a n g e ; I R : i n c i d e n c e r a t e ; SI R : s t an d a r d i z e d i n c i d e n c e r a t e ; VA E R S : V a c c i n e A d v e r s e Ev e n t R e p o r t i n g S y s t e m . SO U R C E S : F o r m e i s t e r e t a l . , 2 0 2 2 ; F r o n t e r a e t a l . , 2 0 2 2 ; L e o n g e t a l . , 2 0 2 3 ; N i e m i n e n e t a l . , 2 0 2 3 ; Y a n i r e t a l . , 2 0 2 2 . Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. HEARING LOSS AND TINNITUS 119 PREPUBLICATION COPY: Uncorrected Proofs Nieminen et al. (2023) compared the incidence rate of SSNHL in the 30-day window preceding vaccination and 0–54 days and more than 54 days after vaccination to that between January 1, 2019, and March 1, 2020, using a national Finnish electronic health database (n = 5.5 million people); excluding those with pre-existing recent diagnosis of SSNHL from the period immediately before the study (2015–2018). Finland’s national vaccination register provided the vaccination dates and product names. SSNHL was identified using an ICD-10 diagnostic code for specialized care visits and from hospital wards. Comorbid conditions were identified from multiple sources. Models were adjusted for calendar time, SARS-CoV-2 infection, demographic, cardiovascular, chronic comorbidities, and health care use covariates. Relative to the incidence before March 2020, adjusted models suggest no increased risk in the initial 0–54-day risk period after the first dose or second dose with BNT162b2 (Dose 1: IR 0.8, 95% CI: 0.6–1.0; Dose 2: IRR 0.8, 95% CI: 0.6–1.2), or mRNA-1273 2 (Dose 1: IR 0.8, 95% CI: 0.5–1.4; Dose 2: IRR 1.2, 95% CI: 0.7–1.9). Secondary models examining risk after 54 days postvaccination and after a third dose likewise yielded no associations. Yanir et al. (2022) used the Clalit Health Services database in Israel to estimate the incidence of SSNHL after first and second doses of BNT162b2 from December 20, 2020, to April 30, 2021. Subsequent analysis compared estimates to the incidence of SSNHL from the same database in 2018 and 2019 and developed age- and sex- standardized incidence ratios. SSNHL was identified using a broad array of ICD-9 codes for hearing loss (388.2, 389.1, 389.10–389.13, 389.15–389.18, 389.8, and 389.9) and concurrent prednisone use within 30 days of diagnosis. The authors reported that 2.6 million people (mean [SD] age, 46.8 [19.6] years; 51.5 percent female) received the first dose, with 91 cases of SSNHL reported. Of these, 2.4 million (93.8 percent) received the second dose, with 79 cases of SSNHL reported. The age- and sex-weighted standardized incidence ratios (SIR) were 1.35 (95% CI: 1.09–1.65) after the first dose and 1.23 (95% CI: 0.98–1.53) after the second dose when using 2018 data as a reference (the sensitivity analysis was similar when using 2019 data). Leong et al. (2023) leveraged a clinical convenience sample from an otology clinic (NYC, NY) (rather than prospective outreach) from May to July 2021 to characterize the incidence of hearing loss after COVID-19 vaccination. Among 500 individuals who completed screening (median age 56.6 years; 59.4 percent female), 420 reported being vaccinated (58.4 percent BNT162b2, 29.1 percent mRNA-1273, 3.3 percent Ad26.COV2.S); 21 (5 percent) reported hearing loss within 4 weeks of vaccination. However, after comprehensive audiologic and otologic evaluation, only seven cases (1.7 percent of vaccinated individuals) were deemed to be SSNHL; the rest represented new or exacerbated symptoms of known pathologies of hearing loss that did not represent SSNHL definition or were unrelated to vaccination. The study did not compare vaccinated to unvaccinated individuals. Despite concerns with selection bias, recall bias and confounding, a key finding from this paper was that self-reported declines in hearing after vaccination may be unreliable, as a majority of cases were attributable to other etiologies. Inaccurate reporting of tinnitus may lead to overestimation of observed associations. Two included studies used data from the U.S. Vaccine Adverse Events Reporting System (VAERS). For denominators, each of these studies utilized publicly available data from the CDC on the total number of individuals vaccinated with COVID-19 vaccines and the total number of doses administered in the United States during the time frames of interest. As part of a larger analysis of neurologic events after COVID-19 vaccination, Frontera et al. (2022) reported an 2 Refers to the COVID-19 vaccine manufactured by Moderna under the name Spikevax®. Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. 120 VACCINE EVIDENCE REVIEW PREPUBLICATION COPY—Uncorrected Proofs incidence rate (IR) of 3.26 cases of hearing loss identified by free text or automated coding per 1,000,000 vaccines (IR per 1,000,000 by vaccine type: 3.20 BNT162b2, 3.08 mRNA-1273, 6.29 Ad26.COV2.S) between January 1, 2021, and June 14, 2021 (306.9 million COVID-19 vaccine doses; 314,610 total adverse events). The number of hearing loss events are not specifically reported (Frontera et al., 2022). Formeister et al. (2022) described the incidence rate of SSNHL in the initial 7-month period of the U.S. vaccination campaign (December 14, 2020, to July 16, 2021). The authors identified 2,170 reports of hearing loss after vaccination in VAERS (search terms: sudden hearing loss, deafness, deafness neurosensory, deafness unilateral, deafness bilateral, and hypoacusis). Of those, the authors deemed 555 events as credible because they occurred within 21 days of vaccination and had one of the following: reference to an audiologic assessment, evaluation by an otolaryngologist, audiologist, or other physician resulting in diagnosis of SSNHL, or evaluation by an otolaryngologist resulting in magnetic resonance imaging and/or treatment with systemic or intratympanic steroid medication. The resultant estimates of annual incidence of SSNHL after COVID-19 vaccination in VAERS data were between 0.6 (probable; minimum estimate) and 28.0 (maximum estimate) cases per 100,000 people per year. The authors note that this is lower than or similar to the estimated annual U.S. incidence (11–77 per 100,000 people per year) (Formeister et al., 2022). In a secondary analysis, the authors note that the reports per 100,000 doses in VAERS decreased from 1.10 in December 2020 to 0.01 in June 2021, despite large increases in the absolute number of vaccines administered. From Evidence to Conclusions The broader academic literature includes a handful of published articles reporting sudden sensorineural hearing loss (SSNHL) in individuals receiving COVID-19 vaccination; however, this level of evidence does not support an association between vaccination and SSNHL (Formeister et al., 2022; Jeong and Choi, 2021; Tsetsos et al., 2021). However, the committee found that the majority of the literature was limited to single case reports, unadjusted descriptive reports lacking a comparison or without thoughtful adjudication of hearing loss, or publications with potential bias and these did not meet our inclusion criteria during screening. Only one of the studies included in this review suggested an association between COVID-19 vaccination and SSNHL. However, the magnitude of the effect was small, with potential for confounding from unmeasured variables. In contrast, the most methodologically rigorous analysis that included potential confounders (e.g., infection status, comorbidities, and health care use patterns) in models found no association. Using pharmacovigilance data without comparators offers low- level evidence to support a conclusion. Nonetheless, one article used VAERS data and offered compelling evidence that incidence of SSNHL were similar to expected rates and much lower after an adjudication procedure to assess the credibility of the reported hearing loss. Moreover, the same report showed that the weekly number of reports of SSNHL did not change over time despite large increases in the number of vaccines administered. An emergent theme is heterogeneity in identification of SSNHL and potential for misclassification. First, self-reported data may be unreliable. Insights from the reviewed literature may reflect this. Formeister et al. (2022) offered insights that many reports of SSNHL in the VAERS data may not be true cases, and Leong et al. (2023) found that the majority of self- reported new cases from vaccination were attributable to exacerbating known etiologies of hearing loss. Another consideration may be that hearing includes peripheral encoding and central Evidence Review of the Adverse Effects of COVID-19 Vaccination and Intramuscular Vaccine Administration Copyright National Academy of Sciences. All rights reserved. HEARING LOSS AND TINNITUS 121 PREPUBLICATION COPY: Uncorrected Proofs processing of information in the brain, and cognitive processes play a key role in how individuals understand speech. The role of cognition in the potential association between self-reported hearing and COVID-19 infection or vaccination may be highly overlooked, as it is plausible that existing age-related hearing loss, which is highly prevalent, could be perceived as “new” due to fatigue or “brain fog.” Second, studies varied in definitions of SSNHL, including using different risk-windows. Moreover, different and unverified approaches to diagnosis codes were used. Two studies used diagnosis codes. Yanir et al. (2022) took a wide approach by looking for many different ICD codes for hearing loss with concurrent prednisone usage; Nieminen et al. (2023) used a single SSNHL code. Given the acute nature of SSNHL, diagnosis codes may be accurate and reliable with some suggestion that an audiological test battery occurred. However, it is unknown if concurrent ICD codes for more general hearing loss paired with prednisone use is reliable. No studies examined the relationship between NVX-CoV2373 3 and SSNHL. Overall, we found that the literature on vaccination and sensorineural hearing loss focused almost exclusively on SSNHL. Our review of said literature resulted in weak evidence and concerns about the measurement of SSNHL. Although the combination of the more methodologically rigorous evidence suggesting no association and lack of identified potential mechanisms beyond hypotheses may hint at no relationship between vaccination and SSNHL, the literature is inadequate to offer a decision on the acceptance or rejection of a causal relationship. Future epidemiological evidence is required. Conclusion 4-1: The evidence is inadequate to accept or reject a causal relationship between the BNT162b2 vaccine and sensorineural hearing loss. Conclusion 4-2: The evidence is inadequate to accept or reject a causal relationship between the mRNA-1273 vaccine and sensorineural hearing loss. Conclusion 4-3: The evidence is inadequate to accept or reject a causal relationship between the Ad26.COV2.S vaccine and sensorineural hearing loss. Conclusion 4-4: The evidence is inadequate to accept or reject a causal relationship between the NVX-CoV2373 vaccine and sensorineural hearing loss. 3 Refers to the COVID-19 vaccine manufactured by Novavax.