Trial reportJournal of neurology2020
'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab.
Trial report in Journal of neurology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 68 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)
A Phase III, Open-label Extension Trial of ECU-MG-301 to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)
Who cites it
68 citing papers in PubMed, 2 syntheses or guidelines pooled it, 89 citations in OpenAlex.
- Efficacy and safety of complement inhibitors and FcRn blockers in generalized AChR antibody-positive myasthenia gravis: a meta-analysis.Journal of neurology · 2026Pooled it
- Assessing the effectiveness of measurement scales in evaluating the health-related quality of life in rare disease patients after treatment: a systematic review.Health and quality of life outcomes · 2024Pooled it
- Evaluation of Sustained Disease Control With Nipocalimab Versus Placebo in the Phase 3 Vivacity-MG3 Study.European journal of neurology · 2026Trial
- Durability of Response to B-Cell Maturation Antigen-Directed mRNA Cell Therapy in Myasthenia Gravis.Annals of clinical and translational neurology · 2025Trial
- Long-Term Efficacy and Safety of Ravulizumab in Adults With Anti-Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: Final Results From the Phase 3 CHAMPION MG Open-Label Extension.European journal of neurology · 2025Trial
- Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study.The Lancet. Neurology · 2023Trial
- Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.Neurology · 2021Trial
- Real-World Clinical Outcomes and Corticosteroid Tapering in Telitacicept-Based Therapy for Ocular Myasthenia Gravis: An Exploratory Analysis by Prior Treatment History.Brain and behavior · 2026Article
- Treatment Preferences of Patients With Myasthenia Gravis: A Qualitative Study.Muscle & nerve · 2026Article
- Extended Preoperative Immunotherapy Duration Reduces the Risk of Myasthenia Gravis Exacerbation for Patients with Thymoma: A Multicenter Retrospective Cohort Study.Neurology and therapy · 2026Article
- Facilitators and barriers to exercise in autoimmune myasthenia gravis: A cross-sectional survey study.Journal of neuromuscular diseases · 2026Article
- Tacrolimus as Single-Agent Immunotherapy for Adult-Onset Myasthenia Gravis: Remission, Relapse, and Safety.CNS neuroscience & therapeutics · 2026Article
- Disease changes and cost analysis of myasthenia gravis: a 10 years retrospective study from a reference center in Italy.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026Article
- Burden of Disease in Myasthenia Gravis: A Targeted Literature Review.Journal of neurology · 2026Review
- Assessing respiratory status in myasthenia gravis: limited value of the MG-ADL as a standalone tool compared with spirometry in a Danish cohort.Journal of neurology · 2026Article
- Minimal Symptom Expression in Generalized Myasthenia Gravis: A Valuable Patient-Centric Treatment Goal.European journal of neurology · 2026Review
- Who Can Safely Discontinue Treatment in Myasthenia Gravis? Insights From a Long-Term Real-World Study.European journal of neurology · 2026Article
- Early versus late add-on therapy in generalized myasthenia gravis: a multicenter real-world cohort study.Journal of neurology · 2026Observational
- Telitacicept as a New Therapeutic Avenue for Generalized Myasthenia Gravis and Thymoma-Associated Myasthenia Gravis.Journal of neuroimmune pharmacology : the official journal of the Society on NeuroImmune Pharmacology · 2026Article
- Myasthenia gravis: from recognition of heterogeneity to a paradigm shift toward precision therapy.Frontiers in immunology · 2026Review
8 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 4 institutions in 3 countries.
Funding
Abstract
backgroundThe 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.
methodsAttainment 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.
resultsAt 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.
conclusionsPatients 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 registrationClinicalTrials.gov NCT01997229, NCT02301624.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.