Trial reportAnnals of clinical and translational neurology2021
Long-term efficacy of eculizumab in refractory generalized myasthenia gravis: responder analyses.
Trial report in Annals of clinical and translational neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
What it found
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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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
35 citing papers in PubMed, 60 citations in OpenAlex.
- Time to response with ravulizumab, a long-acting terminal complement inhibitor, in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.European journal of neurology · 2024Trial
- Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension.Journal of neurology · 2023Trial
- Practical Guidance on Initiating and Switching Targeted Immunotherapies in Generalised Myasthenia Gravis: A German-Austrian Expert Opinion Paper.European journal of neurology · 2026Article
- Experience Using Efgartigimod to Treat Juvenile Myasthenia Gravis in China: A Multicenter Retrospective Study.Muscle & nerve · 2026Article
- Progress in the Treatment of Refractory Myasthenia Gravis.Revista de neurologia · 2026Review
- Cardiopulmonary exercise testing in mild generalized myasthenia gravis: an exploratory pilot feasibility study.Frontiers in physiology · 2026Article
- AChR Autoantibody Pathogenic Properties Are Heterogeneously Distributed and Undergo Temporal Changes Among Patients With Myasthenia Gravis.Neurology(R) neuroimmunology & neuroinflammation · 2025Article
- Immunomodulators and immunosuppressants for myasthenia gravis: a network meta-analysis.The Cochrane database of systematic reviews · 2025Article
- Eculizumab as a Rescue Therapy in Prolonged Myasthenic Crisis in the Intensive Care Unit: A Case Series.Neurocritical care · 2025Article
- Article
- C5 complement inhibition versus FcRn modulation in generalised myasthenia gravis.Journal of neurology, neurosurgery, and psychiatry · 2025Observational
- Ravulizumab and Efgartigimod in Myasthenia Gravis: A Real-World Study.Neurology(R) neuroimmunology & neuroinflammation · 2025Article
- Patterns and predictors of therapeutic response to efgartigimod in acetylcholine receptor-antibody generalized myasthenia gravis subtypes.Therapeutic advances in neurological disorders · 2025Article
- Thymic hyperplasia in myasthenia gravis: a narrative review.Mediastinum (Hong Kong, China) · 2025Review
- Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2024Review
- Real-world experience with eculizumab and switching to ravulizumab for generalized myasthenia gravis.Annals of clinical and translational neurology · 2024Article
- Eculizumab Treatment of Massive Hemolysis Occurring in a Rare Co-Existence of Paroxysmal Nocturnal Hemoglobinuria and Myasthenia Gravis.Hematology reports · 2024Article
- Eculizumab in thymoma-associated myasthenia gravis: a real-world cohort study.Therapeutic advances in neurological disorders · 2024Article
- Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study.Therapeutic advances in neurological disorders · 2024Article
- Complements and Their Role in Systemic Disorders.Cureus · 2024Review
Corrections and comments
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Authors and funding
6 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveGeneralized myasthenia gravis (gMG) is an autoimmune disease that causes disabling weakness via damage to the neuromuscular junction. In most patients, the disease is mediated by autoantibodies to the acetylcholine receptor, which activate the complement cascade. Our objective was to analyze response profiles in adult patients with anti-acetylcholine receptor antibody-positive refractory gMG treated with eculizumab-a terminal complement inhibitor-in the REGAIN study or its open-label extension (OLE).
methodsWe retrospectively analyzed Myasthenia Gravis-Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores recorded during REGAIN and its OLE. Early/late responses were defined as improvement in MG-ADL score (≥3 points) or QMG score (≥5 points) at ≤12 or >12 weeks, respectively, after eculizumab initiation.
resultsThe analysis included 98 patients. By Week 12 and conclusion of the OLE, MG-ADL response had been achieved at some point by 67.3% and 84.7% of patients, respectively, and QMG response by 56.1% and 71.4%, respectively. Response was observed over multiple consecutive assessments for most patients. At Week 130, the least-squares mean percentage changes (95% CI) from baseline in MG-ADL score were -61.9% (-69.9%, -53.9%) and -47.5% (-59.0%, -36.0%) in early and late MG-ADL responders, respectively; the least-squares mean percentage changes from baseline in QMG score were -40.8% (-48.3%, -33.4%) and -55.5% (-68.4%, -42.7%) in early and late QMG responders, respectively.
interpretationThe findings suggest that, although most patients with refractory gMG will achieve clinical response by Week 12 of eculizumab treatment, first responses can be observed with longer-term treatment.
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