@article{70f71444ca15420987001d9f43b76a56,
title = "Long-term safety and efficacy of eculizumab in generalized myasthenia gravis",
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.",
author = "Srikanth Muppidi and Kimiaki Utsugisawa and Michael Benatar and Hiroyuki Murai and Barohn, {Richard J.} and Isabel Illa and Saiju Jacob and John Vissing and Burns, {Ted M.} and Kissel, {John T.} and Nowak, {Richard J.} and Henning Andersen and Carlos Casasnovas and {de Bleecker}, {Jan L.} and Vu, {Tuan H.} and Renato Mantegazza and O'Brien, {Fanny L.} and Wang, {Jing Jing} and Fujita, {Kenji P.} and Howard, {James F.} and {Regain Study Group} and Mazia, {Claudio Gabriel} and Miguel Wilken and Fabio Barroso and Juliet Saba and Marcelo Rugiero and Mariela Bettini and Marcelo Chaves and Gonzalo Vidal and Garcia, {Alejandra Dalila} and {van den Abeele}, Guy and {de Koning}, Kathy and {de Mey}, Katrien and Rudy Mercelis and Mahieu, {D. lphine} and Linda Wagemaekers and {van Damme}, Philip and Annelies Depreitere and Caroline Schotte and Charlotte Smetcoren and Olivier Stevens and {van Daele}, Sien and Nicolas Vandenbussche and Annelies Vanhee and Sarah Verjans and Jan Vynckier and Ann D'Hondt and Petra Tilkin and {Alves de Siqueira Carvalho}, Alzira and {van der Kooi}, Anneke and {de Visser}, Marianne",
year = "2019",
month = jul,
day = "1",
doi = "https://doi.org/10.1002/mus.26447",
language = "English",
volume = "60",
pages = "14--24",
journal = "Muscle & Nerve",
issn = "0148-639X",
publisher = "John Wiley & Sons, Inc",
number = "1",
}