Eculizumab improves fatigue in refractory generalized myasthenia gravis

The REGAIN Study Group

    Research output: Contribution to journalArticlepeer-review

    18 Citations (Scopus)

    Abstract

    Purpose: To evaluate the effect of eculizumab on perceived fatigue in patients with anti-acetylcholine receptor antibody-positive, refractory, generalized myasthenia gravis (MG) using the Quality of Life in Neurological Disorders (Neuro-QOL) Fatigue subscale, and to evaluate correlations between improvements in Neuro-QOL Fatigue and other clinical endpoints. Methods: Neuro-QOL Fatigue, MG Activities of Daily Living (MG-ADL), Quantitative MG (QMG), and the 15-item MG Quality of Life (MG-QOL15) scales were administered during the phase 3, randomized, placebo-controlled REGAIN study (eculizumab, n = 62; placebo, n = 63) and subsequent open-label extension (OLE). Data were analyzed using repeated-measures models. Correlations between changes in Neuro-QOL Fatigue and in MG-ADL, QMG, and MG-QOL15 scores were determined at REGAIN week 26. Results: At REGAIN week 26, eculizumab-treated patients showed significantly greater improvements in Neuro-QOL Fatigue scores than placebo-treated patients (consistent with improvements in MG-ADL, QMG, and MG-QOL15 scores previously reported in REGAIN). Improvements with eculizumab were sustained through OLE week 52. Correlations between Neuro-QOL Fatigue and MG-QOL15, MG-ADL, and QMG scores were strong for eculizumab-treated patients at REGAIN week 26, and strong, moderate, and weak, respectively, for placebo-treated patients. Conclusions: Compared with placebo, eculizumab was associated with improvements in perceived fatigue that strongly correlated with improvements in MG-specific outcome measures. Trial ID Registration: NCT01997229, NCT02301624.

    Original languageEnglish
    Pages (from-to)2247-2254
    Number of pages8
    JournalQuality of Life Research
    Volume28
    Issue number8
    DOIs
    Publication statusPublished - 2019 Aug 15

    Keywords

    • Complement
    • Eculizumab
    • Fatigue
    • Myasthenia gravis
    • Neuro-QOL Fatigue
    • Quality of life
    • Terminal complement inhibition

    ASJC Scopus subject areas

    • Public Health, Environmental and Occupational Health

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