Mycophenolate mofetil treatment of myasthenia gravis

OBJECTIVE: To review published literature evaluating the effectiveness of mycophenolate mofetil for the treatment of myasthenia gravis (MG). DATA SOURCES: Searches of MEDLINE (1966-August 2005) and Cochrane Database (1993-August 2005) were conducted. Studies conducted in humans and published in English were retrieved. Additional data were identified through subsequent bibliographic reviews. DATA SYNTHESIS: Interruption of T- and B-lymphocyte proliferation in various autoimmune diseases has been investigated. Mycophenolate is known to inhibit lymphocyte proliferation and has shown improved clinical responses in several autoimmune diseases including lupus erythematosus, rheumatoid arthritis, and systemic vasculitis. Data suggesting similar benefits in MG treatment have been reported in case reports, retrospective analyses, an open-label trial, and a randomized, double-blind trial. CONCLUSIONS: Limited evidence from retrospective analyses and clinical trials suggests that mycophenolate is a possible treatment option for patients with MG. Improvement in clinical symptoms and a steroid-sparing effect have been reported when mycophenolate is used in this patient population. Larger, randomized, controlled, and comparative trials are needed to establish optimal dose, time to effect, specific therapeutic role, and long-term safety for mycophenolate when used for treating MG.