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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. [ABSTRACT FROM AUTHOR]

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 (ACT) 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 ACT 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. [ABSTRACT FROM AUTHOR]

Background: We report our experience of patients with generalised myasthenia gravis (gMG) treated with efgartigimod, an neonatal Fc receptor antagonist, under the Early Access to Medicine Scheme (EAMS) in the UK., Methods: Data from all UK patients treated with efgartigimod under the EAMS July 2022 to July 2023 were collected retrospectively. Efgartigimod was administered as per the ADAPT protocol (consisting of a treatment cycle of four infusions at weekly intervals with further cycles given according to clinical need)., Results: 48 patients with acetylcholine receptor antibody-positive gMG were treated in 12 centres. Most (75%) were female and most had a disease duration of over 10 years. The average MG-Activities of Daily Living (ADL) score at baseline was 11.2. Most (72.9%) patients had undergone thymectomy. 77.0% were taking prednisolone at baseline. All patients had used non-steroidal immunosuppressant treatments, the average number tried was 2.6 (range 1-6). 51% had received rituximab. 54.2% of patients required regular intravenous immunoglobulin/plasma exchange.75% of patients had a mean reduction in the MG-ADL of≥2 points in the first cycle and this remained stable throughout the study. The mean intracycle reduction in the MG-ADL score in the first, second, third and fourth cycles were -4.6 to -3.9, -3.4 and -4.2, respectively. Side effects were generally mild. No rescue treatments were required. At the end of the study, 96% of patients remained on efgartigimod., Conclusion: Efgartigimod is a safe and effective treatment for patients with refractory, treatment-resistant gMG., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2025. No commercial re-use. See rights and permissions. Published by BMJ Group.)