Mycophenolate mofetil as second line immunosuppressant in Myasthenia gravis--a long-term prospective open-label study.

The preferred immunosuppressive drug for long term treatment of myasthenia gravis (MG) is azathioprine (AZA). Mycophenolate mofetil (MMF) was suggested as an effective and safe second line alternative to AZA. In a prospective open-label study, 11 patients with acetylcholine receptor antibody (AchR-ab) positive MG (n = 4 ocular MG, n = 7 generalized MG) were treated with MMF which replaced AZA. Reasons for the change of immunosuppressant therapy were side effects (n = 9) or unresponsiveness under AZA (n = 3). Mean duration of MMF treatment was 16.9 months (6-46 months). During MMF treatment AZA side effects resolved in 8/9 patients, concomitant therapy could be discontinued in 4 patients and reduced in 5 patients, and 5 patients remitted and 3 remained in remission. One MMF-refractory patient required add-on IVIG therapy and another with ocular MG showed signs of generalization after 20 months of MG treatment. One patient was diagnosed with bronchial carcinoma after 10 months of MMF treatment. Due to its favourable spectrum of side effects compared to AZA MMF might serve as a second-line immunosuppressant in those MG patients who have not tolerated AZA.