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Eculizumab improves fatigue in refractory generalized myasthenia gravis

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  • Henning Andersen
  • Renato Mantegazza
  • Jing Jing Wang
  • Fanny O'Brien
  • Kaushik Patra
  • James F Howard
  • REGAIN Study Group
  • John Vissing (Member of study group)
  • Nanna Witting (Member of study group)
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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
JournalQuality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation
Volume28
Issue number8
Pages (from-to)2247-2254
Number of pages8
ISSN0962-9343
DOIs
Publication statusPublished - Aug 2019

    Research areas

  • Activities of Daily Living, Antibodies, Monoclonal, Humanized/therapeutic use, Fatigue/drug therapy, Female, Humans, Male, Middle Aged, Myasthenia Gravis/drug therapy, Outcome Assessment (Health Care), Placebos/therapeutic use, Quality of Life, Receptors, Cholinergic/immunology

ID: 58457638