Efficacy of mycophenolate mofetil in the treatment of chronic graft-versus-host disease

Francisco Lopez, Pablo Parker, Auayporn Nademanee, Roberto Rodriguez, Zaid Al-Kadhimi, Ravi Bhatia, Sandra Cohen, Peter Falk, Henry Fung, Mark Kirschbaum, Amrita Krishnan, Neil Kogut, Arturo Molina, Ryotaro Nakamura, Margaret O'Donnell, Leslie Popplewell, Vinod Pullarkat, Joseph Rosenthal, Firoozeh Sahebi, Eileen SmithDavid Snyder, George Somlo, Ricardo Spielberger, Anthony Stein, Robert Sweetman, Jasmine Zain, Stephen Forman

Research output: Contribution to journalArticlepeer-review

69 Scopus citations


Current treatment of chronic graft-versus-host disease (cGVHD) with prednisone (PSE) alone or with added cyclosporine or tacrolimus still has a very high failure and complication rate, and new treatment approaches are needed for both primary and salvage therapy. Mycophenolate mofetil (MMF) is an immunosuppressive agent currently in use for acute graft-versus-host disease prophylaxis. To determine whether MMF had activity in the treatment of cGVHD, we added MMF to standard cyclosporine, tacrolimus, and/or PSE as salvage/second-line (n = 24) or first-line (n = 10) therapy in 34 patients. Nine (90%) of 10 patients receiving first-line and 18 (75%) of 24 receiving second-line MMF therapy responded. Twelve (35%) patients had a complete remission, 15 (44%) had a partial remission, 5 (15%) had stable disease, and only 2 (6%) had progressive disease. Out of 30 patients receiving PSE, 22 (73%) were able to decrease PSE doses (median decrease of 50%; range, 25%-100%). With a median follow-up of 24 months (range, 6-28 months), 29 (85%) patients are alive. Three patients had to discontinue MMF because of abdominal cramps within 3 months of starting treatment. These data suggest that MMF is an active, well-tolerated agent in the treatment of cGVHD and may have a beneficial effect on the survival of patients with this complication.

Original languageEnglish (US)
Pages (from-to)307-313
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Issue number4
StatePublished - Apr 2005
Externally publishedYes


  • Chronic graft-versus-host disease
  • Mycophenolate mofetil
  • Transplantations

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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