+1 Recommend
0 collections
      • Record: found
      • Abstract: found
      • Article: not found

      National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. The 2014 Clinical Trial Design Working Group Report.

      Read this article at

          There is no author summary for this article yet. Authors can add summaries to their articles on ScienceOpen to make them more accessible to a non-specialist audience.


          Treatment of chronic graft-versus-host disease is intended to produce a sustainable benefit by reducing symptom burden, controlling objective manifestations of disease activity, preventing damage and impairment, and improving overall survival without causing disproportionate harms related to the treatment itself. Successful management can control the disease until systemic treatment is no longer needed. The complexity of the disease, the extended duration of follow-up needed to observe disease resolution and withdrawal of immunosuppressive treatment, and the lack of fully developed shorter term endpoints impede progress in the field. Identification and characterization of primary endpoints demonstrating clinical benefit without requiring years of follow-up is urgently needed, with the understanding that clinical benefit encompasses not only the self-evident benefit of the primary endpoint but also any other associated benefits. This report discusses regulatory considerations, eligibility criteria, the value of controlled trial designs, the merits of proposed primary endpoints, and key considerations elaborated from experience and progress during the past decade. The report concludes by mapping an overall approach that could support and lead to maximally informative clinical trials, especially those that seek to demonstrate clinical benefit along a pathway to regulatory review and approval.

          Related collections

          Author and article information

          Biol. Blood Marrow Transplant.
          Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation
          Elsevier BV
          Aug 2015
          : 21
          : 8
          [1 ] Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington. Electronic address:
          [2 ] Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
          [3 ] Center for Drug Evaluation Research, US Food and Drug Administration, Silver Spring, Maryland.
          [4 ] Division of Hematology and Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin.
          [5 ] Division of Hematologic Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts.
          [6 ] Department of Oncology, Johns Hopkins University School of Medicine, Baltimore, Maryland.
          [7 ] Department of Medicine, McMaster University, Hamilton, Ontario, Canada.
          [8 ] Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland.
          [9 ] Stem Cell Transplant Program, Banner MD Anderson Cancer Center, Gilbert, Arizona.
          [10 ] Clinical Hematology and Cellular Therapy Department, Hôpital Saint-Antoine, University Pierre & Marie Curie, INSERM U938, Paris, France.
          [11 ] Department of Internal Medicine III, University of Regensburg, Regensburg, Germany.
          [12 ] Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
          S1083-8791(15)00327-4 NIHMS692066


          Comment on this article