Biology of Blood and Marrow Transplantation
Volume 12, Issue 5 , Pages 491-505, May 2006

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

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
    • Corresponding Author InformationCorrespondence and reprint requests: Paul J. Martin, MD, Fred Hutchinson Cancer Research Center, PO Box 19024, 1100 Fairview Avenue N, D2-100, Seattle, WA 98109-1024
    • ,
  • Daniel Weisdorf

      Affiliations

    • University of Minnesota, Minneapolis, Minnesota
    • ,
  • Donna Przepiorka

      Affiliations

    • University of Tennessee, Memphis, Tennessee
    • ,
  • Steven Hirschfeld

      Affiliations

    • US Food and Drug Administration, Rockville, Maryland
    • ,
  • Ann Farrell

      Affiliations

    • US Food and Drug Administration, Rockville, Maryland
    • ,
  • J. Douglas Rizzo

      Affiliations

    • Medical College of Wisconsin, Milwaukee, Wisconsin
    • ,
  • Ronan Foley

      Affiliations

    • McMaster University, Hamilton, Ontario, Canada
    • ,
  • Gerard Socie

      Affiliations

    • Hopital St. Louis, Paris, France
    • ,
  • Shelly Carter

      Affiliations

    • EMMES Corporation, Rockville, Maryland
    • ,
  • Daniel Couriel

      Affiliations

    • University of Texas M.D. Anderson Cancer Center, Houston, Texas
    • ,
  • Kirk R. Schultz

      Affiliations

    • British Columbia Children’s Hospital, University of British Columbia, Vancouver, British Columbia, Canada
    • ,
  • Mary E.D. Flowers

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington
    • ,
  • Alexandra H. Filipovich

      Affiliations

    • Cincinnati Children’s Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio
    • ,
  • Rima Saliba

      Affiliations

    • University of Texas M.D. Anderson Cancer Center, Houston, Texas
    • ,
  • Georgia B. Vogelsang

      Affiliations

    • Johns Hopkins University School of Medicine, Baltimore, Maryland
    • ,
  • Steven Z. Pavletic

      Affiliations

    • National Cancer Institute, National Institutes of Health, Bethesda, Maryland
    • ,
  • Stephanie J. Lee

      Affiliations

    • Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington

Received 8 March 2006; accepted 9 March 2006.

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.

Key words:  Chronic graft-versus-host disease , Allogeneic hematopoietic cell transplantation , Consensus , Clinical trials , Design

 

 The opinions expressed here are those of the authors and do not represent the official position of the National Institutes of Health, US Food and Drug Administration, or the US Government.

PII: S1083-8791(06)00250-3

doi:10.1016/j.bbmt.2006.03.004

Biology of Blood and Marrow Transplantation
Volume 12, Issue 5 , Pages 491-505, May 2006

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