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Podcast: Long-term Safety and Efficacy of Eculizumab in Generalized Myasthenia Gravis

12/18/2019- Dr. Elahi Behzad interviews Dr. Srikanth Muppidi on his article, Long-term Safety and Efficacy of Eculizumab in Generalized Myasthenia Gravis. Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy. Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001). These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.    27 minutes

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