Please use this identifier to cite or link to this item: http://hdl.handle.net/2445/196128
Title: Consistent improvement with eculizumab across muscle groups in myasthenia gravis
Author: Mantegazza, Renato
O'Brien, Fanny L.
Yountz, Marcus
Howard, James F. Jr.
REGAIN Study Group
Casasnovas Pons, Carlos
Keywords: Anticossos monoclonals
Malalties neuromusculars
Malalties autoimmunitàries
Monoclonal antibodies
Neuromuscular diseases
Autoimmune diseases
Issue Date: Jun-2020
Publisher: American Neurological Association
Abstract: 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.
Note: Reproducció del document publicat a: https://doi.org/10.1002/acn3.51121
It is part of: Annals of Clinical and Translational Neurology, 2020, vol. 7, num. 8, p. 1327-1339
URI: http://hdl.handle.net/2445/196128
Related resource: https://doi.org/10.1002/acn3.51121
ISSN: 2328-9503
Appears in Collections:Articles publicats en revistes (Ciències Clíniques)

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