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'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab
Journal article   Open access   Peer reviewed

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

John Vissing, Saiju Jacob, Kenji P Fujita, Fanny O'Brien, James F Howard and REGAIN study group
Journal of neurology, Vol.267(7), pp.1991-2001
07/2020
DOI: 10.1007/s00415-020-09770-y
PMCID: PMC7320935
PMID: 32189108
url
https://doi.org/10.1007/s00415-020-09770-yView
Published (Version of record) Open Access

Abstract

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. 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. 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. 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. ClinicalTrials.gov NCT01997229, NCT02301624.
 Quality of Life Activities of Daily Living Adult Aged Antibodies, Monoclonal, Humanized - administration & dosage Antibodies, Monoclonal, Humanized - adverse effects Antibodies, Monoclonal, Humanized - pharmacology Autoantibodies Double-Blind Method Female Humans Immunologic Factors - administration & dosage Immunologic Factors - adverse effects Immunologic Factors - pharmacology Male Middle Aged Myasthenia Gravis - drug therapy Myasthenia Gravis - immunology Myasthenia Gravis - physiopathology Patient Reported Outcome Measures Receptors, Cholinergic - immunology

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