Therapy with mycophenolate mofetil for refractory acute and chronic GVHD.

Publication Type:

Journal Article


Bone marrow transplantation, Volume 44, Issue 11, p.739-48 (2009)


2009, Administration, Oral, Adolescent, Adult, Center-Authored Paper, Child, Child, Preschool, Clinical Research Division, Female, Graft vs Host Disease, Humans, Immunosuppressive Agents, Male, Middle Aged, Mycophenolic Acid, Prospective Studies, Research Trials Office Core Facility - Biostatistics Service, Retrospective Studies, Shared Resources, Specimen Processing Core Facility, Treatment Outcome, Young Adult


We evaluated the pharmacokinetics and efficacy of oral mycophenolate mofetil (MMF) for treatment of refractory GVHD. In a prospective study of acute GVHD, 9 of 19 patients (47%) had a response and 10 (53%) had no improvement. Survival at 6 and 12 months after the start of MMF was 37 and 16%, respectively. In a retrospective study of acute GVHD, 14 of 29 patients (48%) had a response and 15 (52%) had no improvement. Survival at 6 and 12 months was 55 and 52%, respectively. In a prospective study of chronic GVHD, the cumulative incidence of disease resolution and withdrawal of all systemic immunosuppressive treatment was 9, 17 and 26% at 12, 24 and 36 months, respectively, after starting MMF. Thirteen patients (59%) required additional systemic immunosuppressive treatment for chronic GVHD. Nine of the 42 patients (21%) in the prospective studies discontinued MMF treatment because of toxicity. The area under the curve plasma concentrations of mycophenolic acid seemed to be suboptimal among patients with acute GVHD but not among those with chronic GVHD. MMF can be used effectively for treatment of GVHD.