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

Biol Blood Marrow Transplant. 2006 May;12(5):491-505. doi: 10.1016/j.bbmt.2006.03.004.


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.

Publication types

  • Consensus Development Conference, NIH
  • Guideline
  • Research Support, N.I.H., Intramural
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Adrenal Cortex Hormones / administration & dosage
  • Adrenal Cortex Hormones / therapeutic use
  • Adult
  • Child
  • Chronic Disease
  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / standards
  • Clinical Trials, Phase II as Topic / methods
  • Clinical Trials, Phase III as Topic / methods
  • Data Collection
  • Disease Progression
  • Forecasting
  • Forms and Records Control
  • Graft vs Host Disease / drug therapy
  • Graft vs Host Disease / prevention & control
  • Graft vs Host Disease / therapy*
  • Humans
  • Immunosuppression Therapy* / adverse effects
  • Immunosuppression Therapy* / methods
  • Immunosuppressive Agents / administration & dosage
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use
  • Medical Records
  • Multicenter Studies as Topic
  • Patient Selection
  • Patients / psychology
  • Research Design
  • Treatment Outcome
  • United States
  • United States Food and Drug Administration / standards


  • Adrenal Cortex Hormones
  • Immunosuppressive Agents