Real-world effectiveness and safety of zilucoplan in patients with anti-AChR myasthenia gravis: A retrospective cohort study in France - PubMed
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- #myasthenia gravis
- #zilucoplan
- #real-world evidence
- The study evaluated zilucoplan's effectiveness and safety in a real-world setting for 48 patients with anti-AChR myasthenia gravis in France.
- Patients showed significant improvement in MG-ADL scores, decreasing from 7.4 at baseline to 2.5 at six months, indicating reduced symptom burden.
- Minimal symptom expression was achieved by 9 out of 43 patients at three months and 12 out of 30 at six months.
- Muscle strength (MMS-Garches) and quality of life (MG-QoL15r) scores significantly improved over six months, with p-values <0.0001 and 0.0156 respectively.
- Adverse events occurred in 15 patients, leading to discontinuation in four cases, but no major adverse events were reported.
- Oral corticosteroid use decreased from an average of 28.8 mg to 17.9 mg, and MG crises reduced from 22 in the prior six months to 6 during treatment.
- The study concluded that zilucoplan is effective in improving symptoms, reducing crises and corticosteroid exposure, and is generally well-tolerated.