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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. Biol Blood Marrow Transplant 2018 Jun;24(6):1274-1280 PMID: 29325830 PMCID: PMC5993573

Pubmed ID



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.

Author List

Pasquini MC, Logan B, Jones RJ, Alousi AM, Appelbaum FR, BolaƱos-Meade J, Flowers MED, Giralt S, Horowitz MM, Jacobsohn D, Koreth J, Levine JE, Luznik L, Maziarz R, Mendizabal A, Pavletic S, Perales MA, Porter D, Reshef R, Weisdorf D, Antin JH


Mary M. Horowitz MD, MS Center Director, Professor in the Medicine department at Medical College of Wisconsin
Marcelo C. Pasquini MD, MS Associate Professor in the Medicine department at Medical College of Wisconsin

jenkins-FCD Prod-296 4db9d02597e0a2e889e230f853b641c12f1c3ee3