Research
Print page Print page
Switch language
The Capital Region of Denmark - a part of Copenhagen University Hospital
Published

Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Research output: Contribution to journalJournal articleResearchpeer-review

DOI

  1. Translational challenges of remote ischemic conditioning in ischemic stroke - a systematic review

    Research output: Contribution to journalReviewResearchpeer-review

  2. Intravenous arylsulfatase A in metachromatic leukodystrophy: a phase 1/2 study

    Research output: Contribution to journalJournal articleResearchpeer-review

  3. Habitual sleep disturbances and migraine: a Mendelian randomization study

    Research output: Contribution to journalJournal articleResearchpeer-review

  4. The impact of gender, puberty, and pregnancy in patients with POLG disease

    Research output: Contribution to journalJournal articleResearchpeer-review

  5. Responsiveness of outcome measures in myotonic dystrophy type 1

    Research output: Contribution to journalJournal articleResearchpeer-review

  • REGAIN Study Group
View graph of relations

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.

Original languageEnglish
JournalAnnals of Clinical and Translational Neurology
Volume7
Issue number8
Pages (from-to)1327 - 1339
Number of pages13
ISSN2328-9503
DOIs
Publication statusPublished - 22 Jul 2020
Externally publishedYes

ID: 61072192