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

• Published in: Therapeutic advances in neurological disorders Vol. 18; p. 17562864251332037
• Main Authors: Marini, Sofia, Falso, Silvia, Habetswallner, Federico, Marini, Martina, Iorio, Raffaele
• Format: Journal Article
• Language: English
• Published: England SAGE Publications 01.01.2025; SAGE Publishing
• Subjects: Brief Report; myasthenia gravis; steroid-sparing agents; corticosteroids; complement inhibitors; acetylcholine receptor antibodies
• ISSN: 1756-2864; 1756-2856; 1756-2864
• DOI: 10.1177/17562864251332037

Using complement inhibitor therapy to reduce steroid doses in patients with generalized myasthenia gravis This study evaluated the effectiveness of complement inhibitor therapy in reducing steroid use in patients with generalized myasthenia gravis (gMG), an autoimmune disease associated with autoantibodies targeting the neuromuscular junction, leading to muscle weakness and fatigability. Standard treatment for gMG involves corticosteroids and non-steroidal immunosuppressants such as azathioprine and mycophenolate mofetil. However, long-term steroid use can lead to significant systemic adverse effects. Recently approved targeted therapies, including complement inhibitors such as eculizumab and ravulizumab, offer a promising approach by inhibiting the complement C5 protein. However, there is limited real-world data on their ability to reduce steroid use. In this study, we enrolled 69 gMG patients who were receiving steroids at baseline, in addition to either azathioprine, mycophenolate mofetil, or complement inhibitors. We assessed changes in steroid dosage at different time points. Our results showed a significant reduction in steroid use across all treatment groups. Notably, patients treated with complement inhibitors experienced a more significant reduction in steroid dosage at earlier time points, compared to those receiving azathioprine or mycophenolate mofetil. These findings suggest that complement inhibitors may be effective in reducing steroid use in gMG patients, making them a promising treatment option for minimizing steroid doses and their associated side effects.