Long‐term safety and efficacy of eculizumab in generalized myasthenia gravis. Issue 1 (8th March 2019)

• Record Type: Journal Article
• Authors: Muppidi, Srikanth; Utsugisawa, Kimiaki; Benatar, Michael; Murai, Hiroyuki; Barohn, Richard J.; Illa, Isabel; Jacob, Saiju; Vissing, John; Burns, Ted M.; Kissel, John T.; Nowak, Richard J.; Andersen, Henning; Casasnovas, Carlos; de Bleecker, Jan L.; Vu, Tuan H.; Mantegazza, Renato; O'Brien, Fanny L.; Wang, Jing Jing; Fujita, Kenji P.; Howard, James F.
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• Is Part Of: Muscle & nerve. Volume 60:Issue 1(2019)
• Page Start: 14
• Page End: 24
• Publication Date: 2019-03-08
• Subjects: eculizumab -- MG‐ADL -- MGC -- MG‐QOL15 -- myasthenia gravis -- QMG; Neuromuscular diseases -- Periodicals; Muscles -- Periodicals; Nerves -- Periodicals; 616.74
• DOI: 10.1002/mus.26447
• Languages: English
• ISSNs: 0148-639X

Abstract

ABSTRACT: Introduction : 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. Methods : Eculizumab (1, 200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results : 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). Discussion : These findings provide evidence for the long‐term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019 Abstract : See editorial on pages 7–9 in this issue .