Abstract
Preventing chronic graft-versus-host disease (GVHD) remains challenging because the unique cellular and molecular pathways that incite chronic GVHD are poorly understood. One major point of intervention for potential prevention of chronic GVHD occurs at the time of transplantation when acute donor anti-recipient immune responses first set the events in motion that result in chronic GVHD. After transplantation, additional insults causing tissue injury can incite aberrant immune responses and loss of tolerance, further contributing to chronic GVHD. Points of intervention are actively being identified so that chronic GVHD initiation pathways can be targeted without affecting immune function. The major objective in the field is to continue basic studies and to translate what is learned about etiopathology to develop targeted prevention strategies that decrease the risk of morbid chronic GVHD without increasing the risks of cancer relapse or infection. Development of strategies to predict the risk of developing debilitating or deadly chronic GVHD is a high research priority. This working group recommends further interrogation into the mechanisms underpinning chronic GVHD development, and we highlight considerations for future trial design in prevention trials.
| Original language | English |
|---|---|
| Pages (from-to) | 452-466 |
| Number of pages | 15 |
| Journal | Transplantation and Cellular Therapy |
| Volume | 27 |
| Issue number | 6 |
| DOIs | |
| State | Published - Jun 2021 |
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In: Transplantation and Cellular Therapy, Vol. 27, No. 6, 06.2021, p. 452-466.
Research output: Contribution to journal › Review article › peer-review
TY - JOUR
T1 - National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease
T2 - I. The 2020 Etiology and Prevention Working Group Report
AU - Williams, Kirsten M.
AU - Inamoto, Yoshihiro
AU - Im, Annie
AU - Hamilton, Betty
AU - Koreth, John
AU - Arora, Mukta
AU - Pusic, Iskra
AU - Mays, Jacqueline W.
AU - Carpenter, Paul A.
AU - Luznik, Leo
AU - Reddy, Pavan
AU - Ritz, Jerome
AU - Greinix, Hildegard
AU - Paczesny, Sophie
AU - Blazar, Bruce R.
AU - Pidala, Joseph
AU - Cutler, Corey
AU - Wolff, Daniel
AU - Schultz, Kirk R.
AU - Pavletic, Steven Z.
AU - Lee, Stephanie J.
AU - Martin, Paul J.
AU - Socie, Gerard
AU - Sarantopoulos, Stefanie
N1 - Funding Information: Financial disclosure: Funding and implementation of this Consensus Project have been made possible through the support of the Intramural Program of the National Cancer Institute Center for Cancer Research, the National Institute of Dental and Craniofacial Research, and the National Institutes of Health intramural and extramural research programs, institutes, and centers. Special acknowledgment goes to the Meredith Cowden GVHD foundation, France Lymphome Espoir, NBMTLink; Anthony Nolan; National Marrow Donor Program; BMT InfoNet; and other patient advocacy groups for partnering and collaboration. Thanks also go to all working groups and consensus conference participants, professional societies, US government agencies, and stakeholders in the field of hematopoietic stem cell transplantation for the generous donation of their work, time, talents, and expertise. We particularly acknowledge the American Society for Transplantation and Cellular Therapy and European Society for Blood and Marrow Transplantation for their roles in the dissemination, education, and implementation of the concepts and best practices evolving from this project. The opinions expressed are those of the authors and do not represent the position of the National Cancer Institute, the National Institutes of Health, or the US Government. Funding Information: Special thanks to the independent external peer reviewers who provided comments and critiques to the 2020 NIH Chronic GVHD Consensus Project: Nicolaus Kroeger, MD, Professor and Clinical Director of the Department of Stem Cell Transplantation, University of Hamburg, Hamburg, Germany, and EBMT president. Ryotaro Nakamura, MD, Professor and Director of the Center for Stem Cell Transplantation City of Hope Cancer Center, Duarte, California. John DiPersio, MD, PhD, Chief, Division of Oncology; Director, Center for Gene and Cellular Immunotherapy; Deputy Director, Siteman Cancer Center, Washington University School of Medicine, St. Louis, Missouri. Mark Juckett, MD, Professor and Director of the Blood and Marrow Transplant Program, University of Wisconsin, Madison, Wisconsin. George Chen, MD, Associate Professor of Medicine, University at Buffalo, Buffalo, New York. Rafael Duarte, MD, PhD, FRCP, Head of the Department of Hematology and Director of the Hematopoietic Transplant Program, Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain. Franco Locatelli, MD, Professor of Pediatrics Università Sapienza, Roma, Head of the Department of Pediatric Hematology and Cell and Gene Therapy, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy. Areej El-Jawahri, MD, Associate Professor, Director of the Bone Marrow Transplant Survivorship Program, and Associate Director of the Cancer Outcomes Research and Education Program at Massachusetts General Hospital, Boston, Massachusetts. Robert Soiffer, MD, Professor, Chair of the Executive Committee for Clinical Programs, Vice Chair for the Department of Medical Oncology, Chief of the Division of Hematologic Malignancies, Dana Farber Cancer Institute, Boston, Massachusetts. Daniel Weisdorf, MD, Professor of Medicine and Deputy Director of Clinical Science and Translational Science Institute; Director, Clinical and Translational Research Services, University of Minnesota, Minneapolis, Minnesota. Keith Sullivan, MD, Professor of Medicine, Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, North Carolina. Catherine Lee, MD, Assistant Professor, Hematologic malignancies, Huntsman Cancer Institute–University of Utah, Salt Lake City, Utah. Jose Antonio Perez-Simon, MD, Professor of Hematology, University of Seville, Head of Department of Hematology, University Hospital Virgen del Rocio and Vice Director of the Biomedical Research Institute of Seville, Seville, Spain. Doris Ponce, MD, hematologist–oncologist, Associate Professor of Medicine, Memorial Sloan-Kettering Cancer Center, New York City, New York. Andrew Harris, MD, pediatric hematologist–oncologist and Assistant Professor of Pediatrics, Pediatric BMT and Cellular Therapy Program, University of Utah/Primary Children's Hospital, Salt Lake City, Utah. Financial disclosure: Funding and implementation of this Consensus Project have been made possible through the support of the Intramural Program of the National Cancer Institute Center for Cancer Research, the National Institute of Dental and Craniofacial Research, and the National Institutes of Health intramural and extramural research programs, institutes, and centers. Special acknowledgment goes to the Meredith Cowden GVHD foundation, France Lymphome Espoir, NBMTLink; Anthony Nolan; National Marrow Donor Program; BMT InfoNet; and other patient advocacy groups for partnering and collaboration. Thanks also go to all working groups and consensus conference participants, professional societies, US government agencies, and stakeholders in the field of hematopoietic stem cell transplantation for the generous donation of their work, time, talents, and expertise. We particularly acknowledge the American Society for Transplantation and Cellular Therapy and European Society for Blood and Marrow Transplantation for their roles in the dissemination, education, and implementation of the concepts and best practices evolving from this project. The opinions expressed are those of the authors and do not represent the position of the National Cancer Institute, the National Institutes of Health, or the US Government. Conflict of interest statement: K.M.W. A.I. J.W.M. P.R. H.G. and S.Z.P. have no conflicts of interest to report. Y.I. has served on advisory boards for Novartis, Janssen, and Meiji Seika Pharma. B.H. has served on the advisory board for Syndax. J.K. has received research support from Amgen, BMS, Clinigen, Equillium, Miltenyi Biotec, and Regeneron Pharmaceuticals; has served on the advisory boards for Cugene and Therakos; and has received consulting income from Biolojic Design, EMD Serono, Equillium, Gentibio, Moderna, and Nekonal Oncology. M.A. has served as a consultant for Fate Therapeutics and has received research funding from Pharmacyclics, Kadmon, and Syndax. I.P. has served on the advisory boards for Incyte, Kadmon, and Syndax. P.A.C. has been a consultant for Fate Therapeutics. L.L. receives research support from Genentech and Merck; has served on the advisory boards for AbbVie, Talaris Therapeutics, and PrecisionBiosciences; and is a patent holder for WindMIL Therapeutics. J.R. has received research funding from Amgen, Equillium, and Kite Pharma; serves on the Data Monitoring Committee for Avrobio; and has received consulting income from BMS/Celgene, Infinity Pharmaceuticals, LifeVault Bio, Rheos Medicines, Talaris Therapeutics, and TScan Therapeutics. S.P. has patent applications (US 20130115232A1 and WO2013066369A3) on “Methods of detection of graft versus host disease” licensed to ViaCore-IBT laboratories. B.R.B. has served on the advisory boards for Magenta Therapeutics and BlueRock Therapeutics; has received research funding from BlueRock Therapeutics; has served on the steering committee for Kadmon Corporation; and is the co-founder of Tmunity Therapeutics. J.P. has consulted and served on the advisory boards for Syndax, CTI Biopharma, and Amgen, Regeneron and has received clinical trial support from Novartis, Amgen, Takeda, Janssen, Johnson & Johnson, Pharmacyclics, Abbvie, CTI Biopharma, and BMS. C.C. has consulted for and received honoraria from Incyte, Jazz, CareDx, Mesoblast, Syndax, Omeros, and Pfizer. D.W. has served on the advisory boards for Novartis and Incyte; has served on data and safety monitoring boards for Novartis and Behring; and has received honoraria from Takeda, Gilead, Pfizer, and Neovii. K.R.S. has served on the data and safety monitoring board for BMS/Juno and on the advisory board for Jazz, Novartis, and Janssen. S.J.L. has received research funding from Amgen, AstraZeneca, Incyte, Kadmon, Novartis, Pfizer, Syndax, and Takeda and has served on the steering committee for Incyte. P.J.M. has served on the advisory boards for Mesoblast and Rigel Pharmaceuticals, Inc. and has received honoraria from Janssen. G.S. has served on the advisory boards for Novartis, Incyte, Pharmacyclics, Amgen, and Xenikos. S.S. has served on the advisory board for Rigel Pharmaceuticals, Inc. Financial disclosure: See Acknowledgments on page 462. Publisher Copyright: © 2021 The American Society for Transplantation and Cellular Therapy
PY - 2021/6
Y1 - 2021/6
N2 - Preventing chronic graft-versus-host disease (GVHD) remains challenging because the unique cellular and molecular pathways that incite chronic GVHD are poorly understood. One major point of intervention for potential prevention of chronic GVHD occurs at the time of transplantation when acute donor anti-recipient immune responses first set the events in motion that result in chronic GVHD. After transplantation, additional insults causing tissue injury can incite aberrant immune responses and loss of tolerance, further contributing to chronic GVHD. Points of intervention are actively being identified so that chronic GVHD initiation pathways can be targeted without affecting immune function. The major objective in the field is to continue basic studies and to translate what is learned about etiopathology to develop targeted prevention strategies that decrease the risk of morbid chronic GVHD without increasing the risks of cancer relapse or infection. Development of strategies to predict the risk of developing debilitating or deadly chronic GVHD is a high research priority. This working group recommends further interrogation into the mechanisms underpinning chronic GVHD development, and we highlight considerations for future trial design in prevention trials.
AB - Preventing chronic graft-versus-host disease (GVHD) remains challenging because the unique cellular and molecular pathways that incite chronic GVHD are poorly understood. One major point of intervention for potential prevention of chronic GVHD occurs at the time of transplantation when acute donor anti-recipient immune responses first set the events in motion that result in chronic GVHD. After transplantation, additional insults causing tissue injury can incite aberrant immune responses and loss of tolerance, further contributing to chronic GVHD. Points of intervention are actively being identified so that chronic GVHD initiation pathways can be targeted without affecting immune function. The major objective in the field is to continue basic studies and to translate what is learned about etiopathology to develop targeted prevention strategies that decrease the risk of morbid chronic GVHD without increasing the risks of cancer relapse or infection. Development of strategies to predict the risk of developing debilitating or deadly chronic GVHD is a high research priority. This working group recommends further interrogation into the mechanisms underpinning chronic GVHD development, and we highlight considerations for future trial design in prevention trials.
UR - http://www.scopus.com/inward/record.url?scp=85103581487&partnerID=8YFLogxK
U2 - 10.1016/j.jtct.2021.02.035
DO - 10.1016/j.jtct.2021.02.035
M3 - Review article
C2 - 33877965
AN - SCOPUS:85103581487
SN - 2666-6367
VL - 27
SP - 452
EP - 466
JO - Transplantation and Cellular Therapy
JF - Transplantation and Cellular Therapy
IS - 6
ER -