Martin, Paul J. and Lee, Stephanie J. and Przepiorka, Donna and Horowitz, Mary M. and Koreth, John and Vogelsang, Georgia B. and Walker, Irwin and Carpenter, Paul A. and Griffith, Linda M. and Akpek, Gorgun and Mohty, Mohamad and Wolff, Daniel and Pavletic, Steven Z. and Cutler, Corey S. (2015) 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. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 21 (8). pp. 1343-1359. ISSN 1083-8791, 1523-6536
Full text not available from this repository. (Request a copy)Abstract
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. (C) 2015 American Society for Blood and Marrow Transplantation.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | SYSTEMIC-LUPUS-ERYTHEMATOSUS; MEASURING THERAPEUTIC RESPONSE; REFRACTORY CHRONIC GVHD; FAILURE-FREE SURVIVAL; ACTIVITY INDEX; EXTRACORPOREAL PHOTOPHERESIS; MARROW-TRANSPLANTATION; PRELIMINARY DEFINITION; RHEUMATOID-ARTHRITIS; AMERICAN-COLLEGE; Chronic graft-versus-host disease; Allogeneic hematopoietic cell transplantation; Clinical trials; Design; Consensus; Guidelines |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie) |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 03 Jul 2019 11:03 |
| Last Modified: | 03 Jul 2019 11:03 |
| URI: | https://pred.uni-regensburg.de/id/eprint/5128 |
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