Endpoints for Clinical Trials Testing Treatment of Acute Graft-versus-Host Disease: A Joint Statement

Paul J. Martin, Carlos R. Bachier, Hans Georg Klingemann, Philip L. McCarthy, Paul Szabolcs, Joseph P. Uberti, Michael W. Schuster, Daniel J Weisdorf, Nelson J. Chao, Partow Kebriaei, Elizabeth J. Shpall, Margaret L MacMillan, Robert J. Soiffer

Research output: Contribution to journalArticlepeer-review

47 Scopus citations


Currently, no agents are approved by the United States Food and Drug Administration (FDA) for either prevention or treatment of acute graft-versus-host disease (aGVHD). Formal precedents establishing a comparative basis for assessing the efficacy and safety of new investigational agents are still lacking. As a step toward addressing this problem, a panel of experts met on 2 occasions to reach consensus on recommendations for terminology describing a clinically meaningful primary endpoint in studies assessing treatment for aGVHD. The panel recommended terminology for "very good partial response" (VGPR) that includes both diagnostic and functional criteria. The central hypothesis leading to this proposal is that the potential harm of giving more treatment than needed to produce or maintain complete response exceeds the harm of slight undertreatment that may be associated with less than complete response. VGPR clearly cannot be used as the sole outcome measure in GVHD treatment trials, and must be considered in the context of survival and safety. The proposed use of VGPR as the primary endpoint in GVHD treatment trials will remain provisional until its use has been validated through experience.

Original languageEnglish (US)
Pages (from-to)777-784
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Issue number7
StatePublished - Jul 2009

Bibliographical note

Funding Information:
P.J.M., C.R.B., and M.W.S., research support from Osiris Therapeutics; H.-G.K, independent safety monitor for a clinical trial sponsored by Osiris Therapeutics; D.W., research support from Amgen, Hospira, Easai, Roche, Genzyme, and honoraria for advisory board participation from Genzyme, Dor, Hospira, and Osiris; R.J.S., P.L.M., P.S., J.P.U., N.J.C., P.W., E.J.S., and M.L.M., no additional relevant financial disclosures.

Funding Information:
Financial disclosure: The consensus meetings were held on September 11, 2007, and on May 23, 2008, and were sponsored by Osiris Therapeutics, Inc., Columbia, MD. Osiris provided honoraria to the participants (except P.J.M.), but did not contribute to the meeting output or to the content of this manuscript. Effort by P.J.M. was supported by grant CA18029 from the National Institutes of Health, Department of Health and Human Services.


  • Acute graft-versus-host disease
  • Clinical trials
  • Endpoints
  • Guidelines

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