title: Endpoints for clinical trials testing treatment of acute graft-versus-host disease: a joint statement.
abstract: 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.
authors.affiliation: Fred Hutchinson Cancer Research Center, Seattle, WA 98109-1024, USA. pmartin@fhcrc.org
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Martin
authors.forename: Carlos R
authors.initials: CR
authors.lastname: Bachier
authors.forename: Hans-Georg
authors.initials: HG
authors.lastname: Klingemann
authors.forename: Philip L
authors.initials: PL
authors.lastname: McCarthy
authors.forename: Paul
authors.initials: P
authors.lastname: Szabolcs
authors.forename: Joseph P
authors.initials: JP
authors.lastname: Uberti
authors.forename: Michael W
authors.initials: MW
authors.lastname: Schuster
authors.forename: Daniel
authors.initials: D
authors.lastname: Weisdorf
authors.forename: Nelson J
authors.initials: NJ
authors.lastname: Chao
authors.forename: Partow
authors.initials: P
authors.lastname: Kebriaei
authors.forename: Elizabeth J
authors.initials: EJ
authors.lastname: Shpall
authors.forename: Margaret L
authors.initials: ML
authors.lastname: Macmillan
authors.forename: Robert J
authors.initials: RJ
authors.lastname: Soiffer
mesh_terms: D000208:Acute Disease; D002986:Clinical Trials as Topic / Q000379:methods*; D023381:Endpoint Determination / Q000379:methods*; D006086:Graft vs Host Disease / Q000628:therapy*; D006801:Humans