TY - JOUR
T1 - Consistent improvement with eculizumab across muscle groups in myasthenia gravis
AU - Mantegazza, Renato
AU - O'Brien, Fanny L.
AU - Yountz, Marcus
AU - Howard, James F.
AU - Gabriel Mazia, Claudio
AU - Wilken, Miguel
AU - Barroso, Fabio
AU - Saba, Juliet
AU - Rugiero, Marcelo
AU - Bettini, Mariela
AU - Chaves, Marcelo
AU - Vidal, Gonzalo
AU - Dalila Garcia, Alejandra
AU - De Bleecker, Jan
AU - Van den Abeele, Guy
AU - de Koning, Kathy
AU - De Mey, Katrien
AU - Mercelis, Rudy
AU - Mahieu, Délphine
AU - Wagemaekers, Linda
AU - Van Damme, Philip
AU - Depreitere, Annelies
AU - Schotte, Caroline
AU - Smetcoren, Charlotte
AU - Stevens, Olivier
AU - Van Daele, Sien
AU - Vandenbussche, Nicolas
AU - Vanhee, Annelies
AU - Verjans, Sarah
AU - Vynckier, Jan
AU - D’Hont, Ann
AU - Tilkin, Petra
AU - Alves de Siqueira Carvalho, Alzira
AU - Dias Brockhausen, Igor
AU - Feder, David
AU - Ambrosio, Daniel
AU - César, Pamela
AU - Paula Melo, Ana
AU - Martins Ribeiro, Renata
AU - Rocha, Rosana
AU - Bezerra Rosa, Bruno
AU - Garibaldi, Matteo
AU - Emilio Alboini, Paolo
AU - Iorio, Raffaele
AU - Antozzi, Carlo
AU - Bonanno, Silvia
AU - Locatelli, Alberta
AU - Maggi, Lorenzo
AU - Pasanisi, Maria
AU - Campanella, Angela
N1 - Funding Information:
This work was funded by Alexion Pharmaceuticals. We thank the patients who took part in REGAIN and the OLE trial, as well as their families. We thank our investigators and collaborators for their contributions to the completion of the study. We also thank Sivani Paskaradevan (Alexion Pharmaceuticals) for critical review of the manuscript and Cindy Lane (formerly of Alexion Pharmaceuticals) for clinical study oversight. We acknowledge Douglas Holden, BSc (Oxford PharmaGenesis, Oxford, UK), who provided medical writing support in the production of this manuscript (funded by Alexion Pharmaceuticals).
Publisher Copyright:
© 2020 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association
Copyright:
Copyright 2020 Elsevier B.V., All rights reserved.
PY - 2020/8/1
Y1 - 2020/8/1
N2 - Objective: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. Methods: Patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open-label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open-label extension were analyzed. Results: Of the 125 patients who participated in REGAIN, 117 enrolled in the open-label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open-label extension. Interpretation: Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.
AB - Objective: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. Methods: Patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open-label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open-label extension were analyzed. Results: Of the 125 patients who participated in REGAIN, 117 enrolled in the open-label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open-label extension. Interpretation: Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.
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U2 - 10.1002/acn3.51121
DO - 10.1002/acn3.51121
M3 - Article
C2 - 32700461
AN - SCOPUS:85088297453
VL - 7
SP - 1327
EP - 1339
JO - Annals of Clinical and Translational Neurology
JF - Annals of Clinical and Translational Neurology
SN - 2328-9503
IS - 8
ER -