Abstract: Objective: The therapeutic options currently available for treating refractory graft-vs.-host disease (GVHD) are limited. Therefore, the present study evaluated the efficacy of mycophenolate mofetil (MMF) as a salvage treatment for acute and chronic GVHD in allograft patients.
Methods: Twenty-six consecutive patients with refractory acute (13 patients) or chronic (13 patients) GVHD were enrolled. The first-line treatment for all patients with acute GVHD consisted of a combination of cyclosporine A (CyA) and steroids, while the first-line treatment for chronic GVHD was steroids with or without CyA according to the risk group. MMF was added at a dose of 1.5 or 2 g daily and the steroids were tapered in the refractory cases.
Results: Four (30.8%) of 13 patients with refractory acute GVHD responded to MMF. When analyzing the overall results according to the type of acute GVHD, improvement was observed in four (30.8%) of 13 skin cases, four (44.4%) of nine liver cases, and two (22.9%) of nine gut cases. Ten (76.9%) of 13 patients with refractory chronic GVHD responded to MMF. The common side effects were gastrointestinal disturbance (26.9%) or infectious complications (23.1%). The estimated 2-yr survival rate for patients with acute GVHD and chronic GVHD was 33.3% and 53.9%, respectively.
Conclusions: MMF would appear to be effective and safe for treating refractory chronic GVHD, yet not as effective for treating refractory acute gut GVHD. Accordingly, a prospective randomized clinical trial is warranted to assess the impact of MMF in the treatment of refractory GVHD.