Mycophenolate mofetil may be effective in CNS sarcoidosis but not in sarcoid myopathy

Objective: To describe effectiveness, steroid-sparing effect, and tolerance of the antiproliferative immunosuppressant mycophenolate mofetil (MMF) in neurosarcoidosis. Methods: We describe a retrospective case series of 10 consecutive patients with a diagnosis of neurosarcoidosis who were treated with MMF, alone or in association with corticosteroids, in our teaching hospital. Results: At the time of our study, the mean duration of MMF treatment was 21 months. All but one patient with CNS involvement (n = 8) were in remission (except for hormonal dysfunction) which was complete in 6 patients. MMF was efficient as single-agent induction therapy in one patient. The 3 patients who received MMF as a maintenance therapy after initial response to corticosteroids did not relapse even though steroids were stopped. Out of 4 subjects who demonstrated insufficient response to prior therapy including corticosteroids and immunosuppressive agents, 3 demonstrated significant clinical and radiologic improvement. However, the 2 patients who presented muscular sarcoidosis did not respond to MMF. Among patients treated with steroids at MMF introduction and after excluding those with sarcoid myopathy, the mean dose of corticosteroids was 6 mg/day at the end of the follow-up while it was 59 mg/day at the initiation of MMF. No significant side effects were observed. Conclusions: These data suggest that MMF is effective in CNS sarcoidosis but not in sarcoid myopathy, with a corticosteroid sparing effect and a better tolerance profile than other immunosuppressive agents. Neurology (R) 2011;76:1168-1172