Along with corticosteroids, immunosuppressant drugs are mainstays of disease-modifying therapy for myasthenia gravis (MG). However, their efficacies and optimum use are unclear. We identified seven randomised controlled trials (RCT) of immunosuppressants in generalised MG that qualified for Cochrane Review: (1) azathioprine plus initial prednisolone versus prednisolone; (2) azathioprine plus prednisolone versus prednisolone plus placebo; (3) ciclosporin versus placebo (4) ciclosporin plus prednisolone versus prednisolone plus placebo; (5) cyclophosphamide plus prednisolone versus prednisolone plus placebo; (6) mycophenolate mofetil (MMF) alone or plus either ciclosporin or prednisolone versus placebo alone or plus either ciclosporin or prednisolone; (7) tacrolimus plus corticosteroids with or without plasma exchange versus corticosteroids with or without plasma exchange. All trials were small (14 to 41 participants) and their designs heterogeneous.
The RCT evidence, albeit limited, was that ciclosporin (alone or with corticosteroids) or cyclophosphamide (with corticosteroids) improved MG significantly within 1 year compared with placebo. There was no clear evidence of benefit for azathioprine, MMF, or tacrolimus within 1 year. Larger, better-designed, longer trials are needed.
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Competing interests: IKH was involved in a trial of mycophenolate mofetil in myasthenia gravis sponsored by Aspreva Pharmaceuticals.