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.

journal_name

Blood

journal_title

Blood

authors

Martin PJ,Storer BE,Rowley SD,Flowers ME,Lee SJ,Carpenter PA,Wingard JR,Shaughnessy PJ,DeVetten MP,Jagasia M,Fay JW,van Besien K,Gupta V,Kitko C,Johnston LJ,Maziarz RT,Arora M,Jacobson PA,Weisdorf D

doi

10.1182/blood-2009-02-202937

subject

Has Abstract

pub_date

2009-05-21 00:00:00

pages

5074-82

issue

21

eissn

0006-4971

issn

1528-0020

pii

blood-2009-02-202937

journal_volume

113

pub_type

杂志文章,多中心研究,随机对照试验

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