Long-term safety and efficacy of eculizumab in generalized myasthenia gravis

for the Regain Study Group

    Research output: Contribution to journalArticlepeer-review

    60 Citations (Scopus)

    Abstract

    Introduction: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy. Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results: The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001). Discussion: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019.

    Original languageEnglish
    Pages (from-to)14-24
    Number of pages11
    JournalMuscle and Nerve
    Volume60
    Issue number1
    DOIs
    Publication statusPublished - 2019 Jul

    Keywords

    • MG-ADL
    • MG-QOL15
    • MGC
    • QMG
    • eculizumab
    • myasthenia gravis

    ASJC Scopus subject areas

    • Physiology
    • Clinical Neurology
    • Cellular and Molecular Neuroscience
    • Physiology (medical)

    Fingerprint

    Dive into the research topics of 'Long-term safety and efficacy of eculizumab in generalized myasthenia gravis'. Together they form a unique fingerprint.

    Cite this