Faculty Collaboration Database

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

Date

04/26/2006

Pubmed ID

16635784

DOI

10.1016/j.bbmt.2006.03.004

Scopus ID

2-s2.0-33745447476 (requires institutional sign-in at Scopus site)   160 Citations

Abstract

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.

Author List

Martin PJ, Weisdorf D, Przepiorka D, Hirschfeld S, Farrell A, Rizzo JD, Foley R, Socie G, Carter S, Couriel D, Schultz KR, Flowers ME, Filipovich AH, Saliba R, Vogelsang GB, Pavletic SZ, Lee SJ, Design of Clinical Trials Working Group

Author

J. Douglas Rizzo MS, MD Director, Center Associate Director, Professor in the Medicine department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Adrenal Cortex Hormones
Adult
Child
Chronic Disease
Clinical Trials as Topic
Clinical Trials, Phase II as Topic
Clinical Trials, Phase III as Topic
Data Collection
Disease Progression
Forecasting
Forms and Records Control
Graft vs Host Disease
Humans
Immunosuppressive Agents
Medical Records
Multicenter Studies as Topic
Patient Selection
Patients
Research Design
Treatment Outcome
United States
United States Food and Drug Administration