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

2005; Elsevier BV; Volume: 11; Issue: 4 Linguagem: Inglês

10.1016/j.bbmt.2005.01.011

1523-6536

Francisco López, Pablo Parker, Auayporn Nademanee, Roberto Rodríguez, Zaid Al‐Kadhimi, Ravi Bhatia, Sandra Cohen, Peter Falk, Henry C. Fung, Mark Kirschbaum, Amrita Krishnan, Neil Kogut, Arturo Molina, Ryotaro Nakamura, Margaret O’Donnell, Leslie Popplewell, Vinod Pullarkat, Joseph Rosenthal, Firoozeh Sahebi, Eileen Smith, David S. Snyder, George Somlo, Ricardo Spielberger, Anthony S. Stein, Robert Sweetman, Jasmine Zain, Stephen J. Forman,

Mesenchymal stem cell research

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.