title: Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease.
abstract: We conducted a double-blind, randomized multicenter trial to determine whether the addition of mycophenolate mofetil (MMF) improves the efficacy of initial systemic treatment of chronic graft-versus-host disease (GVHD). The primary endpoint was resolution of chronic GVHD and withdrawal of all systemic treatment within 2 years, without secondary treatment. Enrollment of 230 patients was planned, providing 90% power to observe a 20% difference in success rates between the 2 arms. The study was closed after 4 years because the interim estimated cumulative incidence of success for the primary endpoint was 23% among 74 patients in the MMF arm and 18% among 77 patients in the control arm, indicating a low probability of positive results for the primary endpoint after completing the study as originally planned. Analysis of secondary endpoints showed no evidence of benefit from adding MMF to the systemic regimen first used for treatment of chronic GVHD. The estimated hazard ratio of death was 1.99 (95% confidence interval, 0.9-4.3) among patients in the MMF arm compared with the control arm. MMF should not be added to the initial systemic treatment regimen for chronic GVHD. This trial was registered at www.clinicaltrials.gov as #NCT00089141 on August 4, 2004.
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: Barry E
authors.initials: BE
authors.lastname: Storer
authors.forename: Scott D
authors.initials: SD
authors.lastname: Rowley
authors.forename: Mary E D
authors.initials: ME
authors.lastname: Flowers
authors.forename: Stephanie J
authors.initials: SJ
authors.lastname: Lee
authors.forename: Paul A
authors.initials: PA
authors.lastname: Carpenter
authors.forename: John R
authors.initials: JR
authors.lastname: Wingard
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Shaughnessy
authors.forename: Marcel P
authors.initials: MP
authors.lastname: DeVetten
authors.forename: Madan
authors.initials: M
authors.lastname: Jagasia
authors.forename: Joseph W
authors.initials: JW
authors.lastname: Fay
authors.forename: Koen
authors.initials: K
authors.lastname: van Besien
authors.forename: Vikas
authors.initials: V
authors.lastname: Gupta
authors.forename: Carrie
authors.initials: C
authors.lastname: Kitko
authors.forename: Laura J
authors.initials: LJ
authors.lastname: Johnston
authors.forename: Richard T
authors.initials: RT
authors.lastname: Maziarz
authors.forename: Mukta
authors.initials: M
authors.lastname: Arora
authors.forename: Pamala A
authors.initials: PA
authors.lastname: Jacobson
authors.forename: Daniel
authors.initials: D
authors.lastname: Weisdorf
mesh_terms: D002423:Cause of Death; D002908:Chronic Disease; D004311:Double-Blind Method; D005260:Female; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods; D006801:Humans; D017567:Karnofsky Performance Status; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives*; D015996:Survival Rate; D016896:Treatment Outcome
chemical_list: D009173:Mycophenolic Acid