Consistent improvement with eculizumab across muscle groups in myasthenia gravis
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‐a...
| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
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Wiley
2020-08-01
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| Series: | Annals of Clinical and Translational Neurology |
| Online Access: | https://doi.org/10.1002/acn3.51121 |
| _version_ | 1819134655962546176 |
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| author | Renato Mantegazza Fanny L. O'Brien Marcus Yountz James F. Howard Jr for the REGAIN study group |
| author_facet | Renato Mantegazza Fanny L. O'Brien Marcus Yountz James F. Howard Jr for the REGAIN study group |
| author_sort | Renato Mantegazza |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-22T10:06:39Z |
| format | Article |
| id | doaj.art-c91aa02c322b4018a21dac8c7dc88f75 |
| institution | Directory Open Access Journal |
| issn | 2328-9503 |
| language | English |
| last_indexed | 2024-12-22T10:06:39Z |
| publishDate | 2020-08-01 |
| publisher | Wiley |
| record_format | Article |
| series | Annals of Clinical and Translational Neurology |
| spelling | doaj.art-c91aa02c322b4018a21dac8c7dc88f752022-12-21T18:29:56ZengWileyAnnals of Clinical and Translational Neurology2328-95032020-08-01781327133910.1002/acn3.51121Consistent improvement with eculizumab across muscle groups in myasthenia gravisRenato Mantegazza0Fanny L. O'Brien1Marcus Yountz2James F. Howard Jr3for the REGAIN study groupNeuroimmunology and Neuromuscular Diseases Unit Fondazione IRCCS Istituto Neurologico Carlo Besta Milan ItalyAlexion Pharmaceuticals Boston MAAlexion Pharmaceuticals Boston MADepartment of Neurology University of North Carolina Chapel Hill NCAbstract 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.https://doi.org/10.1002/acn3.51121 |
| spellingShingle | Renato Mantegazza Fanny L. O'Brien Marcus Yountz James F. Howard Jr for the REGAIN study group Consistent improvement with eculizumab across muscle groups in myasthenia gravis Annals of Clinical and Translational Neurology |
| title | Consistent improvement with eculizumab across muscle groups in myasthenia gravis |
| title_full | Consistent improvement with eculizumab across muscle groups in myasthenia gravis |
| title_fullStr | Consistent improvement with eculizumab across muscle groups in myasthenia gravis |
| title_full_unstemmed | Consistent improvement with eculizumab across muscle groups in myasthenia gravis |
| title_short | Consistent improvement with eculizumab across muscle groups in myasthenia gravis |
| title_sort | consistent improvement with eculizumab across muscle groups in myasthenia gravis |
| url | https://doi.org/10.1002/acn3.51121 |
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