title: Measuring therapeutic response in chronic graft-versus-host disease: National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. Response Criteria Working Group report.
abstract: The lack of standardized criteria for quantitative measurement of therapeutic response in clinical trials poses a major obstacle for the development of new agents in chronic graft-versus-host disease (GVHD). This consensus document was developed to address several objectives for response criteria to be used in chronic GVHD-related clinical trials. The proposed measures should be practical for use both by transplantation and nontransplantation medical providers, adaptable for use in adults and in children, and focused on the most important chronic GVHD manifestations. The measures should also give preference to quantitative, rather than semiquantitative, measures; capture information regarding signs, symptoms, and function separately from each other; and use validated scales whenever possible to demonstrate improved patient outcomes and meet requirements for regulatory approval of novel agents. Based on these criteria, we propose a set of measures to be considered for use in clinical trials, and forms for data collection are provided (). Measures should be made at 3-month intervals and whenever major changes are made in treatment. Provisional definitions of complete response, partial response, and progression are proposed for each organ and for overall outcomes. The proposed response criteria are based on current expert consensus opinion and are intended to improve consistency in the conduct and reporting of chronic GVHD trials, but their use remains to be demonstrated in practice.
authors.affiliation: National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892-1203, USA. pavletis@mail.nih.gov
authors.forename: Steven Z
authors.initials: SZ
authors.lastname: Pavletic
authors.forename: Paul
authors.initials: P
authors.lastname: Martin
authors.forename: Stephanie J
authors.initials: SJ
authors.lastname: Lee
authors.forename: Sandra
authors.initials: S
authors.lastname: Mitchell
authors.forename: David
authors.initials: D
authors.lastname: Jacobsohn
authors.forename: Edward W
authors.initials: EW
authors.lastname: Cowen
authors.forename: Maria L
authors.initials: ML
authors.lastname: Turner
authors.forename: Gorgun
authors.initials: G
authors.lastname: Akpek
authors.forename: Andrew
authors.initials: A
authors.lastname: Gilman
authors.forename: George
authors.initials: G
authors.lastname: McDonald
authors.forename: Mark
authors.initials: M
authors.lastname: Schubert
authors.forename: Ann
authors.initials: A
authors.lastname: Berger
authors.forename: Peter
authors.initials: P
authors.lastname: Bross
authors.forename: Jason W
authors.initials: JW
authors.lastname: Chien
authors.forename: Daniel
authors.initials: D
authors.lastname: Couriel
authors.forename: J P
authors.initials: JP
authors.lastname: Dunn
authors.forename: Jane
authors.initials: J
authors.lastname: Fall-Dickson
authors.forename: Ann
authors.initials: A
authors.lastname: Farrell
authors.forename: Mary E D
authors.initials: ME
authors.lastname: Flowers
authors.forename: Hildegard
authors.initials: H
authors.lastname: Greinix
authors.forename: Steven
authors.initials: S
authors.lastname: Hirschfeld
authors.forename: Lynn
authors.initials: L
authors.lastname: Gerber
authors.forename: Stella
authors.initials: S
authors.lastname: Kim
authors.forename: Robert
authors.initials: R
authors.lastname: Knobler
authors.forename: Peter A
authors.initials: PA
authors.lastname: Lachenbruch
authors.forename: Frederick W
authors.initials: FW
authors.lastname: Miller
authors.forename: Barbara
authors.initials: B
authors.lastname: Mittleman
authors.forename: Esperanza
authors.initials: E
authors.lastname: Papadopoulos
authors.forename: Susan K
authors.initials: SK
authors.lastname: Parsons
authors.forename: Donna
authors.initials: D
authors.lastname: Przepiorka
authors.forename: Michael
authors.initials: M
authors.lastname: Robinson
authors.forename: Michael
authors.initials: M
authors.lastname: Ward
authors.forename: Bryce
authors.initials: B
authors.lastname: Reeve
authors.forename: Lisa G
authors.initials: LG
authors.lastname: Rider
authors.forename: Howard
authors.initials: H
authors.lastname: Shulman
authors.forename: Kirk R
authors.initials: KR
authors.lastname: Schultz
authors.forename: Daniel
authors.initials: D
authors.lastname: Weisdorf
authors.forename: Georgia B
authors.initials: GB
authors.lastname: Vogelsang
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D002986:Clinical Trials as Topic / Q000592:standards; D015975:Consensus Development Conferences, NIH as Topic; D005260:Female; D006086:Graft vs Host Disease / Q000473:pathology* / Q000628:therapy*; D006801:Humans; D008297:Male; D008499:Medical Records / Q000592:standards; D012720:Severity of Illness Index; D013997:Time Factors; D016896:Treatment Outcome; D014481:United States