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Biol Blood Marrow Transplant. 2018 Jun;24(6):1274-1280. doi: 10.1016/j.bbmt.2018.01.002. Epub 2018 Jan 8.

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.

Author information

1
Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin. Electronic address: mpasquini@mcw.edu.
2
Center for International Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, Wisconsin.
3
The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Johns Hopkins Hospital, Baltimore, Maryland.
4
Department of Stem Cell Transplantation, Division of Cancer Medicine, University of Texas M.D. Anderson Cancer Center, Houston, Texas.
5
Fred Hutchinson Cancer Research Center, Seattle, Washington.
6
Memorial Sloan Kettering Cancer Center, New York, New York.
7
Division of Blood and Marrow Transplantation Center for Cancer and Blood Disorders, Children's National Health System, Washington, DC.
8
Dana-Farber Cancer Institute, Boston, Massachusetts.
9
Blood and Marrow Transplant Program, Icahn School of Medicine at Mount Sinai, New York, New York.
10
Adult Blood and Marrow Stem Cell Transplant Program, Knight Cancer Institute, Oregon Health and Science University, Portland, Oregon.
11
The Emmes Corporation, Rockville, Maryland.
12
Experimental Transplantation and Immunology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland.
13
University of Pennsylvania, Philadelphia, Pennsylvania.
14
Blood and Marrow Transplantation Program and Columbia Center for Translational Immunology, Columbia University Medical Center, New York, New York.
15
Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota, Minneapolis, Minnesota.

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.

KEYWORDS:

Clinical trials; GVHD; Hematopoietic cell transplantation

PMID:
29325830
PMCID:
PMC5993573
DOI:
10.1016/j.bbmt.2018.01.002
[Indexed for MEDLINE]
Free PMC Article

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