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 Journal Article
| Authors: | Pasquini, M. C.; Logan, B.; Jones, R. J.; Alousi, A. M.; Appelbaum, F. R.; Bolaños-Meade, J.; Flowers, M. E. D.; Giralt, S.; Horowitz, M. M.; Jacobsohn, D.; Koreth, J.; Levine, J. E.; Luznik, L.; Maziarz, R.; Mendizabal, A.; Pavletic, S.; Perales, M. A.; Porter, D.; Reshef, R.; Weisdorf, D.; Antin, J. H. |
| Article Title: | 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 |
| 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. © 2018 The American Society for Blood and Marrow Transplantation |
| Keywords: | cancer survival; treatment failure; overall survival; comparative study; disease free survival; methotrexate; bortezomib; cyclophosphamide; chronic myeloid leukemia; acute graft versus host disease; chronic graft versus host disease; myeloablative conditioning; nonhodgkin lymphoma; systematic review; etanercept; graft versus host reaction; reduced intensity conditioning; gvhd; tacrolimus; bone marrow transplantation; chronic lymphatic leukemia; cd34 selection; clinical trials; pentostatin; thymocyte antibody; recurrence free survival; hematopoietic cell transplantation; clinical trial (topic); acute myeloid leukemia; maraviroc; human; article |
| Journal Title: | Biology of Blood and Marrow Transplantation |
| Volume: | 24 |
| Issue: | 6 |
| ISSN: | 1083-8791 |
| Publisher: | Elsevier Inc. |
| Date Published: | 2018-06-01 |
| Start Page: | 1274 |
| End Page: | 1280 |
| Language: | English |
| DOI: | 10.1016/j.bbmt.2018.01.002 |
| PROVIDER: | scopus |
| PMCID: | PMC5993573 |
| PUBMED: | 29325830 |
| DOI/URL: | |
| Notes: | Article -- Export Date: 2 July 2018 -- Source: Scopus |
Altmetric
Citation Impact
BMJ Impact Analytics
Related MSK Work