Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease

Paul J. Martin, Barry E. Storer, Scott D. Rowley, Mary E D Flowers, Stephanie J. Lee, Paul A. Carpenter, John R. Wingard, Paul J. Shaughnessy, Marcel P DeVetten, Madan Jagasia, Joseph W. Fay, Koen Van Besien, Vikas Gupta, Carrie Kitko, Laura J. Johnston, Richard T. Maziarz, Mukta Arora, Pamala A. Jacobson, Daniel Weisdorf

Research output: Contribution to journalArticle

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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.

Original languageEnglish (US)
Pages (from-to)5074-5082
Number of pages9
JournalBlood
Volume113
Issue number21
DOIs
StatePublished - Nov 18 2009

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Mycophenolic Acid
Graft vs Host Disease
Grafts
Therapeutics
Multicenter Studies
Hazards
Confidence Intervals
Incidence

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

Martin, P. J., Storer, B. E., Rowley, S. D., Flowers, M. E. D., Lee, S. J., Carpenter, P. A., ... Weisdorf, D. (2009). Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. Blood, 113(21), 5074-5082. https://doi.org/10.1182/blood-2009-02-202937

Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. / Martin, Paul J.; Storer, Barry E.; Rowley, Scott D.; Flowers, Mary E D; Lee, Stephanie J.; Carpenter, Paul A.; Wingard, John R.; Shaughnessy, Paul J.; DeVetten, Marcel P; Jagasia, Madan; Fay, Joseph W.; Van Besien, Koen; Gupta, Vikas; Kitko, Carrie; Johnston, Laura J.; Maziarz, Richard T.; Arora, Mukta; Jacobson, Pamala A.; Weisdorf, Daniel.

In: Blood, Vol. 113, No. 21, 18.11.2009, p. 5074-5082.

Research output: Contribution to journalArticle

Martin, PJ, Storer, BE, Rowley, SD, Flowers, MED, 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 2009, 'Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease', Blood, vol. 113, no. 21, pp. 5074-5082. https://doi.org/10.1182/blood-2009-02-202937
Martin PJ, Storer BE, Rowley SD, Flowers MED, Lee SJ, Carpenter PA et al. Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. Blood. 2009 Nov 18;113(21):5074-5082. https://doi.org/10.1182/blood-2009-02-202937
Martin, Paul J. ; Storer, Barry E. ; Rowley, Scott D. ; Flowers, Mary E D ; Lee, Stephanie J. ; Carpenter, Paul A. ; Wingard, John R. ; Shaughnessy, Paul J. ; DeVetten, Marcel P ; Jagasia, Madan ; Fay, Joseph W. ; Van Besien, Koen ; Gupta, Vikas ; Kitko, Carrie ; Johnston, Laura J. ; Maziarz, Richard T. ; Arora, Mukta ; Jacobson, Pamala A. ; Weisdorf, Daniel. / Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease. In: Blood. 2009 ; Vol. 113, No. 21. pp. 5074-5082.
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