@article{00c2a75059b74b84b41a0b60288658d1,
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.",
keywords = "MG-ADL, MG-QOL15, MGC, QMG, eculizumab, myasthenia gravis",
author = "{for the Regain Study Group} and 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 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 D{\'e}lphine Mahieu 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 {Dias Brockhausen}, Igor and Kim, {Byung Jo}",
note = "Funding Information: The authors thank the patients who took part in REGAIN and the open-label extension study as well as their families; all of the investigators and collaborators for their contributions to the completion of the study; Angela Kaya, PhD, and Gus Khursigara, PhD, (formerly of Alexion Pharmaceuticals) and R{\'o}is{\'i}n Armstrong, PhD, Diaa Diab, MBBS, and Kelley Capocelli, MD, (Alexion Pharmaceuticals) for critical review of the manuscript, and Cindy Lane, MS, (Alexion Pharmaceuticals) for clinical study oversight. We acknowledge Vicky Sanders, PhD, and Ruth Gandolfo, PhD (Oxford PharmaGenesis, Oxford, UK), who provided medical writing support in the production of this manuscript (funded by Alexion Pharmaceuticals). All members of the REGAIN study group are listed in the Supporting Information materials. Publisher Copyright: {\textcopyright} 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.",
year = "2019",
month = jul,
doi = "10.1002/mus.26447",
language = "English",
volume = "60",
pages = "14--24",
journal = "Muscle and Nerve",
issn = "0148-639X",
publisher = "John Wiley and Sons Inc.",
number = "1",
}