Complement inhibitor therapy as a steroid-sparing strategy in generalized myasthenia gravis

补体抑制剂疗法作为重症肌无力全身型治疗中一种减少类固醇用量的策略

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Abstract

BACKGROUND: Standard management of generalized myasthenia gravis associated with anti-acetylcholine receptor autoantibodies (AChR-gMG) includes corticosteroids and nonsteroidal immunosuppressants. Complement inhibitors (CI) represent a more tailored therapeutic strategy. Real-world data on the steroid-sparing efficacy of CI remain limited. OBJECTIVE: To investigate the steroid-sparing efficacy of CI in AChR-gMG. DESIGN: We identified 69 AChR-gMG patients on corticosteroids treated with azathioprine (AZA), mycophenolate mofetil (MMF), or CI. METHODS: Steroid tapering was assessed by comparing corticosteroid dosage at several time-points to baseline. RESULTS: Steroids reductions were statistically significant for all therapies at every time point compared to baseline (all p < 0.001). Pairwise comparisons using the Mann-Whitney U test revealed significant differences between CI and MMF at 3 months (p = 0.0372), 6 months (p = 0.0193), and 9 months (p = 0.0321) and between CI and AZA at 6 months (p = 0.0415). CONCLUSION: CI rapidly and effectively reduced corticosteroid dosage in most AChR-gMG patients, suggesting their potential role as steroid-sparing therapeutic options.

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