ArticleCNS neuroscience & therapeutics2025
Efficacy and Safety of Efgartigimod for Patients With Myasthenia Gravis in a Real-World Cohort of 77 Patients.
Article in CNS neuroscience & therapeutics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Effectiveness and safety of efgartigimod in myasthenia gravis: A meta-analysis of different antibody subtypes.Immunologic research · 2026Pooled it
- Rapid efficacy of efgartigimod for generalized myasthenia gravis patients in acute exacerbations/worsening: multicenter real-world retrospective study.Journal of neurology · 2026Article
- Clinical outcomes, serum IgG changes, and follow-up observations in the treatment of anti-AChR antibody-positive generalized myasthenia gravis with Efgartigimod.BMC neurology · 2026Article
- Efgartigimod-alfa therapy in acetylcholine receptor antibody myasthenia gravis: Romanian experience.Frontiers in neurology · 2026Article
- Real-world study of efgartigimod in AChR antibody-positive generalized myasthenia gravis: thymus status, multi-domain symptom improvement and steroid-sparing effect.Frontiers in immunology · 2026Article
- Research Progress in Pharmacological Targeted Therapy for Myasthenia Gravis.Journal of inflammation research · 2026Review
- Development and External Validation of a Multivariable Model to Predict Early Minimal Symptom Expression Response in Adult Generalized Myasthenia Gravis Patients Treated With Efgartigimod.CNS neuroscience & therapeutics · 2026Article
- Postmarketing adverse events of efgartigimod alfa: a real-world pharmacovigilance study based on the FAERS database.BMJ open · 2025Article
- Efgartigimod for generalized myasthenia gravis: a comprehensive review of clinical evidence and future perspectives.Frontiers in neurologyReview
- Real-world use of efgartigimod in acetylcholine receptor antibody-positive generalized myasthenia gravis: experience from two centers in Greece and Cyprus.Frontiers in neurologyArticle
Corrections and comments
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Authors and funding
13 authors.
Funding
Abstract
aimsEfgartigimod, a first-in-class neonatal Fc receptor antagonist, is approved for generalized myasthenia gravis (gMG). Its safety and efficacy across MG subtypes remain unclear.
methodsThis single-center real-world study (September 2023-July 2024) analyzed patients from an MG registry study in China. The primary efficacy outcome is the mean MG-ADL score changes from baseline at weeks 4, 8, and 12, analyzed via generalized estimating equations. Safety was assessed by adverse events.
resultsAmong 77 patients (mean age 56.1 ± 15.2 years; 59.7% male), 76 completed at least one treatment cycle (20 completed 2 cycles; 1 completed 3 cycles). After efgartigimod treatment, MG-ADL scores decreased significantly by week 4 (mean difference -6.4, 95% CI -7.2 to -5.6, p < 0.001), sustaining through week 12 (-6.9, -7.8 to -6.1, p < 0.001). After the second cycle, MG-ADL scores at week 12 trended lower than the first cycle (mean difference: -0.8, 95% CI: -2.0 to -0.5, p = 0.061). Efficacy was consistent across MGFA classes and thymoma status. In refractory patients, efgartigimod reduced MG-ADL scores (p < 0.001). Adverse events occurred in 3.9% (3/77).
conclusionEfgartigimod safely improved MG-ADL scores and reduced steroid use across MG subtypes, with sustained efficacy through multiple treatment cycles. These findings support its potential when conventional therapies fail.
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