Blood and Marrow Transplant Clinical Trials Network Report on the Development of Novel Endpoints and Selection of Promising Approaches for Graft-versus-Host Disease Prevention Trials

Marcelo C. Pasquini, Brent Logan, Richard J. Jones, Amin M. Alousi, Frederick R. Appelbaum, Javier Bolaños-Meade, Mary E.D. Flowers, Sergio Giralt, Mary M. Horowitz, David Jacobsohn, John Koreth, John E. Levine, Leo Luznik, Richard Maziarz, Adam Mendizabal, Steven Pavletic, Miguel Angel Perales, David Porter, Ran Reshef, Daniel WeisdorfJoseph H. Antin

Research output: Contribution to journalArticlepeer-review

20 Scopus citations

Abstract

Graft-versus-host disease (GVHD) is a common complication after hematopoietic cell transplantation (HCT) and associated with significant morbidity and mortality. Preventing GVHD without chronic therapy or increasing relapse is a desired goal. Here we report a benchmark analysis to evaluate the performance of 6 GVHD prevention strategies tested at single institutions compared with a large multicenter outcomes database as a control. Each intervention was compared with the control for the incidence of acute and chronic GVHD and overall survival and against novel composite endpoints: acute and chronic GVHD, relapse-free survival (GRFS), and chronic GVHD, relapse-free survival (CRFS). Modeling GRFS and CRFS using the benchmark analysis further informed the design of 2 clinical trials testing GVHD prophylaxis interventions. This study demonstrates the potential benefit of using an outcomes database to select promising interventions for multicenter clinical trials and proposes novel composite endpoints for use in GVHD prevention trials.

Original languageEnglish (US)
Pages (from-to)1274-1280
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Volume24
Issue number6
DOIs
StatePublished - Jun 2018

Bibliographical note

Funding Information:
Financial disclosure: Support for this study was provided by Grant #U10HL069294 to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute and the National Cancer Institute , along with contributions by Millennium Pharmaceuticals, Inc. ( BMT CTN 1203 ) and Miltenyi Biotec ( BMT CTN 1301 ). The content is solely the responsibility of the authors and does not necessarily represent the official views of the above-mentioned parties. The CIBMTR is supported primarily by Grant U24-CA76518 from the National Cancer Institute; the National Heart, Lung, and Blood Institute; and the National Institute of Allergy and Infectious Diseases and Grant HHSH234200637015C from the Health Resources and Services Administration, Department of Health and Human Services.

Publisher Copyright:
© 2018 The American Society for Blood and Marrow Transplantation

Keywords

  • Clinical trials
  • GVHD
  • Hematopoietic cell transplantation

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