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

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

来源：Biology of Blood and Marrow Transplantation, 2018, 24(6): 1274-1280.

DOI：10.1016/j.bbmt.2018.01.002

• GVHD
• Clinical trials
• Hematopoietic cell transplantation

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.

• 出版日期2018-6