Eculizumab improves fatigue in refractory generalized myasthenia gravis

Qual Life Res. 2019 Aug;28(8):2247-2254. doi: 10.1007/s11136-019-02148-2. Epub 2019 Mar 23.

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.

Keywords: Complement; Eculizumab; Fatigue; Myasthenia gravis; Neuro-QOL Fatigue; Quality of life; Terminal complement inhibition.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial

MeSH terms

  • 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

Substances

  • Antibodies, Monoclonal, Humanized
  • Placebos
  • Receptors, Cholinergic
  • eculizumab

Associated data

  • ClinicalTrials.gov/NCT01997229
  • ClinicalTrials.gov/NCT02301624