Forskning
Udskriv Udskriv
Switch language
Region Hovedstaden - en del af Københavns Universitetshospital
Udgivet

Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

DOI

  1. Responsiveness of outcome measures in myotonic dystrophy type 1

    Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

  2. Global FKRP Registry: observations in more than 300 patients with Limb Girdle Muscular Dystrophy R9

    Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

  3. No effect of triheptanoin on exercise performance in McArdle disease

    Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

  4. Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints

    Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

  5. Early prediction of phenotypic severity in Citrullinemia Type 1

    Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

  • REGAIN Study Group
Vis graf over relationer

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.

OriginalsprogEngelsk
TidsskriftAnnals of Clinical and Translational Neurology
Vol/bind7/8
Sider (fra-til)1327 - 1339
ISSN2328-9503
DOI
StatusUdgivet - 22 jul. 2020
Eksternt udgivetJa

Bibliografisk note

© 2020 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals LLC on behalf of American Neurological Association.

ID: 61072192