Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Renato Mantegazza; Fanny L O'Brien; Marcus Yountz; James F Howard Jr; REGAIN study group

doi:10.1002/acn3.51121

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Consistent improvement with eculizumab across muscle groups in myasthenia gravis

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Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Renato Mantegazza, Fanny L O'Brien, Marcus Yountz, James F Howard Jr and REGAIN study group

Annals of clinical and translational neurology, Vol.7(8), pp.1327-1339

08/2020

DOI: 10.1002/acn3.51121

PMCID: PMC7448154

PMID: 32700461

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Abstract

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. 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. 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. 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.

Details

Title: Subtitle: Consistent improvement with eculizumab across muscle groups in myasthenia gravis
Creators: Renato Mantegazza - Neuroimmunology and Neuromuscular Diseases Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy
Fanny L O'Brien - Alexion Pharma
Marcus Yountz - Alexion Pharma
James F Howard Jr - Department of Neurology, University of North Carolina, Chapel Hill, NC
REGAIN study group
Contributors: Christopher S Nance (Contributor) - University of Iowa, Neurology
Ludwig Gutmann (Contributor) - University of Iowa, Neurology
Andrea Swenson (Contributor) - University of Iowa, Neurology
Resource Type: Journal article
Publication Details: Annals of clinical and translational neurology, Vol.7(8), pp.1327-1339
DOI: 10.1002/acn3.51121
PMID: 32700461
PMCID: PMC7448154
NLM abbreviation: Ann Clin Transl Neurol
ISSN: 2328-9503
eISSN: 2328-9503
Grant note: DOI: 10.13039/100006396, name: Alexion Pharmaceuticals
Language: English
Date published: 08/2020
Academic Unit: Neurology
Record Identifier: 9984303025202771

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