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

Paul J. Martin, Daniel Weisdorf, Donna Przepiorka, Steven Hirschfeld, Ann Farrell, J. Douglas Rizzo, Ronan Foley, Gerard Socie, Shelly Carter, Daniel Couriel, Kirk R. Schultz, Mary E D Flowers, Alexandra H. Filipovich, Rima Saliba, Georgia B. Vogelsang, Steven Z. Pavletic, Stephanie J. Lee

Research output: Contribution to journalReview articlepeer-review

153 Scopus citations


The complexity of chronic graft-versus-host disease (GVHD) and the lack of established research methods have made it difficult to design, conduct, and analyze clinical trials involving subjects with this disease, even when promising treatment options are available. This consensus document was developed to offer an approach for overcoming these obstacles. Clinical trials in chronic GVHD should adhere to principles of good trial design and practice. Inclusion and exclusion criteria should allow as many subjects to participate as possible without compromising the interpretation of results. Pre-enrollment assessment of chronic GVHD characteristics should be standardized. The protocol should provide clear guidance about administration of study medication and other interventions. Methods of assessing response should be defined and validated in advance. Efficacy endpoints should be selected to reflect clinical benefit. Expert biostatistical support is needed to ensure the validity and reliability of trial results. The use of consistent standards in clinical trial designs to evaluate agents that have activity in pathogenic pathways could facilitate advances in the treatment of chronic GVHD.

Original languageEnglish (US)
Pages (from-to)491-505
Number of pages15
JournalBiology of Blood and Marrow Transplantation
Issue number5
StatePublished - May 2006

Bibliographical note

Funding Information:
This project was supported by the National Institutes of Health’s National Cancer Institute, Office of the Director, Cancer Therapy Evaluation Program, Intramural Research Program and Center for Cancer Research; National Heart Lung and Blood Institute, Division of Blood Diseases and Resources; Office of Rare Diseases, National Institutes of Health, Office of the Director; National Institute of Allergy and Infectious Disease, Transplantation Immunology Branch; and the Health Resources and Services Administration, Division of Transplantation and the Naval Medical Research Center, C. W. Bill Young/Department of Defense Marrow Donor Recruitment and Research Program. The authors also acknowledge the following individuals and organizations that by their participation made this project possible: American Society for Blood and Marrow Transplantation, Center for International Bone and Marrow Transplant Research, Blood and Marrow Transplant Clinical Trials Network, Canadian Blood and Marrow Transplant Group, European Group for Blood and Marrow Transplantation, Pediatric Blood and Marrow Transplant Consortium, and representatives of the South American transplantation centers (Luis F. Bouzas, MD, and Vaneuza Funke, MD). This project was conducted in coordination with the American Society for Clinical Oncology and American Society of Hematology (liaisons, Michael Bishop, MD, and Jeff Coughlin). The organizers are also indebted to patients and patient and research advocacy groups who made this process much more meaningful by their engagement. Special thanks also to Paula Kim who coordinated these efforts.


  • Allogeneic hematopoietic cell transplantation
  • Chronic graft-versus-host disease
  • Clinical trials
  • Consensus
  • Design


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