In 2014, the National Institutes of Health sponsored the second Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease (GVHD). The purpose was to update recommendations about key elements of chronic GVHD research, including definitions for diagnosis, severity scoring, and response measures, based on empirical data published since the first 2005 Consensus Conference. The most significant modifications were to the response assessments, based on studies demonstrating difficulty with the first consensus definitions. The Response Measures Validation Study is a multicenter, prospective cohort study of patients who are starting initial or subsequent treatments for chronic GVHD. The aim of the study is to evaluate the performance of the 2014 response measures and determine whether any other combination of assessments is superior. Clinical data, clinician assessments, patient-reported outcomes, and research samples are collected at enrollment and 3, 6, and 18 months later, and whenever another chronic GVHD systemic treatment is added. The target enrollment of 368 evaluable patients from 12 transplantation centers has been reached. This report describes the rationale, design, and methods of the Chronic GVHD Response Measures Validation Study, and invites other investigators to collaborate with the Consortium to analyze data or specimens.
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Conflict of interest statement : S.J.L. serves on advisory boards for Amgen and Incyte, and has received travel and speaking honoraria from Mallinckrodt. M.J. serves on advisory boards and as a speaker for Janssen and Mallinckrodt, and has received research funding from Janssen and Mallinckrodt. C.C. serves on advisory boards and as a consultant for Bristol-Myers Squibb and Incyte. S.S. serves on advisory boards for Gilead and Pharmacyclics and has received research funding from Gilead. M.F. serves on advisory boards and as a consultant for Pharmacyclics and has received research funding and travel and speaking honoraria from Incyte, Astellas, and Mallinckrodt. The other authors have no conflicts of interest to report.
Financial disclosure: This study was funded by National Cancer Institute (CA118953).
© 2018 The American Society for Blood and Marrow Transplantation
- Allogeneic hematopoietic cell transplantation
- Chronic graft-versus-host disease
- Prospective longitudinal observational study
- Response assessment