National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IIb. The 2020 Preemptive Therapy Working Group Report.

Allogeneic hematopoietic cell transplantation Chronic graft-versus-host disease Consensus Preemptive therapy Risk assignment biomarkers

Journal

Transplantation and cellular therapy
ISSN: 2666-6367
Titre abrégé: Transplant Cell Ther
Pays: United States
ID NLM: 101774629

Informations de publication

Date de publication:
08 2021
Historique:
received: 26 03 2021
accepted: 30 03 2021
pubmed: 10 4 2021
medline: 3 8 2021
entrez: 9 4 2021
Statut: ppublish

Résumé

Chronic graft-versus-host disease (GVHD) commonly occurs after allogeneic hematopoietic cell transplantation (HCT) despite standard prophylactic immune suppression. Intensified universal prophylaxis approaches are effective but risk possible overtreatment and may interfere with the graft-versus-malignancy immune response. Here we summarize conceptual and practical considerations regarding preemptive therapy of chronic GVHD, namely interventions applied after HCT based on evidence that the risk of developing chronic GVHD is higher than previously appreciated. This risk may be anticipated by clinical factors or risk assignment biomarkers or may be indicated by early signs and symptoms of chronic GVHD that do not fully meet National Institutes of Health diagnostic criteria. However, truly preemptive, individualized, and targeted chronic GVHD therapies currently do not exist. In this report, we (1) review current knowledge regarding clinical risk factors for chronic GVHD, (2) review what is known about chronic GVHD risk assignment biomarkers, (3) examine how chronic GVHD pathogenesis intersects with available targeted therapeutic agents, and (4) summarize considerations for preemptive therapy for chronic GVHD, emphasizing trial development, including trial design and statistical considerations. We conclude that robust risk assignment models that accurately predict chronic GVHD after HCT and early-phase preemptive therapy trials represent the most urgent priorities for advancing this novel area of research.

Identifiants

pubmed: 33836313
pii: S2666-6367(21)00820-4
doi: 10.1016/j.jtct.2021.03.029
pmc: PMC8934187
mid: NIHMS1755099
pii:
doi:

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, N.I.H., Intramural

Langues

eng

Sous-ensembles de citation

IM

Pagination

632-641

Subventions

Organisme : CSRD VA
ID : IK2 CX001785
Pays : United States
Organisme : Intramural NIH HHS
ID : ZIA BC011743
Pays : United States

Informations de copyright

Copyright © 2021. Published by Elsevier Inc.

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Auteurs

Joseph Pidala (J)

Blood and Marrow Transplantation and Cellular Immunotherapy, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida. Electronic address: joseph.pidala@moffitt.org.

Carrie Kitko (C)

Division of Pediatric Hematology/Oncology, Dpeartment of Pediatrics, Vanderbilt University Medical Center, Nashville, Tennessee.

Stephanie J Lee (SJ)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

Paul Carpenter (P)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

Geoffrey D E Cuvelier (GDE)

Cancer Care Manitoba, University of Manitoba, Winnipeg, Manitoba, Canada.

Shernan Holtan (S)

Division of Hematology, Oncology, and Transplantation, Department of Medicine, University of Minnesota Medical School, Minneapolis, Minnesota.

Mary E Flowers (ME)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

Corey Cutler (C)

Division of Stem Cell Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, Massachusetts.

Madan Jagasia (M)

Division of Hematology and Oncology, Vanderbilt University Medical Center, Nashville, Tennessee.

Ted Gooley (T)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

Joycelynne Palmer (J)

Division of Biostatistics, Department of Computational and Quantitative Medicine, City of Hope, Duarte, California.

Tim Randolph (T)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

John E Levine (JE)

Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.

Francis Ayuk (F)

Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.

Fiona Dignan (F)

Department of Clinical Haematology, Manchester Royal Infirmary, Manchester University NHS Foundation Trust, Manchester, United Kingdom.

Helene Schoemans (H)

Department of Hematology, University Hospitals Leuven and Department of Public Health, KU Leuven, Leuven, Belgium.

Eric Tkaczyk (E)

Department of Veterans Affairs and Departments of Dermatology and Biomedical Engineering, Vanderbilt University Medical Center, Nashville, Tennessee.

Nosha Farhadfar (N)

Division of Hematology and Oncology, Department of Medicine, University of Florida, Gainesville, Florida.

Anita Lawitschka (A)

Stem Cell Transplantation Unit, St Anna Children's Hospital, Medical University of Vienna, Vienna, Austria; Children's Cancer Research Institute, Vienna, Austria.

Kirk R Schultz (KR)

Pediatric Hematology/Oncology/BMT, BC Children's Hospital, Vancouver, British Columbia, Canada.

Paul J Martin (PJ)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

Stefanie Sarantopoulos (S)

Division of Hematological Malignancies and Cellular Therapy, Duke Cancer Institute, Duke University Department of Medicine, Durham, North Carolina.

Yoshihiro Inamoto (Y)

Department of Hematopoietic Stem Cell Transplantation, National Cancer Center Hospital, Tokyo, Japan.

Gerard Socie (G)

Hematology and Bone Marrow Transplant Department, AP-HP Saint Louis Hospital and University of Paris, Paris, France.

Daniel Wolff (D)

Department of Internal Medicine III, University Hospital of Regensburg, Regensburg, Germany.

Bruce Blazar (B)

Department of Pediatrics, Division of Blood & Marrow Transplantation & Cellular Therapy, University of Minnesota, Minneapolis, Minnesota.

Hildegard Greinix (H)

Clinical Division of Hematology, Medical University of Graz, Graz, Austria.

Sophie Paczesny (S)

Department of Microbiology and Immunology and Department of Pediatrics, Medical University of South Carolina, Charleston, South Carolina.

Steven Pavletic (S)

Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.

Geoffrey Hill (G)

Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.

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