'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab

REGAIN Study Group

Research output: Contribution to journalArticlepeer-review

3 Citations (Scopus)

Abstract

BACKGROUND: The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension.

METHODS: Attainment of 'minimal symptom expression' was evaluated using patient-reported outcome measures of gMG symptoms [MG activities of daily living scale (MG-ADL), 15-item MG quality of life questionnaire (MG-QOL15)] at the completion of REGAIN and during the open-label extension. 'Minimal symptom expression' was defined as MG-ADL total score of 0-1 or MG-QOL15 total score of 0-3.

RESULTS: At REGAIN week 26, more eculizumab-treated patients achieved 'minimal symptom expression' versus placebo [MG-ADL: 21.4% vs 1.7%; difference 19.8%; 95% confidence interval (CI) 8.5, 31.0; p = 0.0007; MG-QOL15: 16.1% vs 1.7%; difference 14.4%; 95% CI 4.3, 24.6; p = 0.0069]. During the open-label extension, the proportion of patients in the placebo/eculizumab group who achieved 'minimal symptom expression' increased after initiating eculizumab treatment and was sustained through 130 weeks of open-label eculizumab (MG-ADL: 1.7 to 27.8%; MG-QOL15: 1.7 to 19.4%). At extension study week 130, similar proportions of patients in the eculizumab/eculizumab and placebo/eculizumab groups achieved 'minimal symptom expression' (MG-ADL: 22.9% and 27.8%, respectively, p = 0.7861; MG-QOL15: 14.3% and 19.4%, respectively, p = 0.7531). The long-term tolerability of eculizumab was consistent with previous reports.

CONCLUSIONS: Patients with AChR+ refractory gMG who receive eculizumab can achieve sustained 'minimal symptom expression' based on patient-reported outcomes. 'Minimal symptom expression' may be a useful tool in measuring therapy effectiveness in gMG.

TRIAL REGISTRATION: ClinicalTrials.gov NCT01997229, NCT02301624.

Original languageEnglish
Pages (from-to)1991-2001
Number of pages11
JournalJournal of Neurology
Volume267
Issue number7
DOIs
Publication statusPublished - Jul 2020

Keywords

  • Activities of Daily Living
  • Adult
  • Aged
  • Antibodies, Monoclonal, Humanized/administration & dosage
  • Autoantibodies
  • Double-Blind Method
  • Female
  • Humans
  • Immunologic Factors/administration & dosage
  • Male
  • Middle Aged
  • Myasthenia Gravis/drug therapy
  • Patient Reported Outcome Measures
  • Quality of Life
  • Receptors, Cholinergic/immunology

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