ArticleFrontiers in neurology2024
Prospective cohort study evaluating efficacy and safety of efgartigimod in Chinese generalized myasthenia gravis patients.
Article in Frontiers in neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers, 2 of them syntheses that pooled it.
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
14 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Effectiveness and safety of efgartigimod in myasthenia gravis: A meta-analysis of different antibody subtypes.Immunologic research · 2026Pooled it
- The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis.European journal of medical research · 2025Pooled it
- Real-world experience with efgartigimod in generalized myasthenia gravis: a single-center retrospective study in China.Frontiers in immunology · 2026Article
- Efgartigimod for Guillain-Barré syndrome: a retrospective analysis of efficacy and safety.Therapeutic advances in neurological disorders · 2026Article
- 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
- Efgartigimod for Generalized Myasthenia Gravis and Beyond: A Narrative Review of Its Pharmacological Profile, Clinical Utility, and Expanding Applications.Biomedicines · 2025Review
- Factors associated with early response to efgartigimod in patients with generalized myasthenia gravis: a multicenter retrospective observational study.Journal of neurology · 2025Observational
- Efgartigimod as a Fast-Acting Add-On Therapy in Antibody-Mediated Autoimmune Encephalitis.European journal of neurology · 2025Article
- Postmarketing adverse events of efgartigimod alfa: a real-world pharmacovigilance study based on the FAERS database.BMJ open · 2025Article
- Real-World Experience with FcRn Inhibitors Efgartigimod and Rozanolixizumab in Myasthenia Gravis: Administration in Multiple Cycles and Transition from Intravenous to Subcutaneous Formulation.Neurology and therapy · 2025Article
- New and Emerging Biological Therapies for Myasthenia Gravis: A Focussed Review for Clinical Decision-Making.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2025Review
- Patterns and predictors of therapeutic response to efgartigimod in acetylcholine receptor-antibody generalized myasthenia gravis subtypes.Therapeutic advances in neurological disorders · 2025Article
- Efgartigimod non-responders after the first treatment cycle in generalized myasthenia gravis: a retrospective analysis of predictive factors.Frontiers in neurology · 2025Article
- Review
Corrections and comments
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Authors and funding
14 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Background: Despite the efficacy of efgartigimod demonstrated in ADAPT phase 3 trial, data specifically derived from Chinese participants are not available. Therefore, we aimed to evaluate the efficacy and safety of efgartigimod in Chinese patients with generalized myasthenia gravis (gMG). Methods: This is a prospective cohort study conducted in 8 hospitals across China. gMG patients received weekly intravenous infusions of efgartigimod (10 mg/kg) under a named patient program (NPP). The present study is an 8-week study, consisting of 4 consecutive doses of efgartigimod administered over 3 weeks (one cycle), followed by a 5-week follow-up period to assess the tolerability of efgartigimod's therapeutic effects. The primary outcome was the mean change in MG activities of daily living (MG-ADL) total score from baseline to 4 weeks. MG-ADL responder was defined as a ≥ 2-point improvement that persisted for 4 weeks, starting by week 4. Safety evaluations encompassed the monitoring of adverse events (AE) and serious AE (SAE) throughout the study. Results: Between 5 July 2022 and 25 August 2023, a total of 14 gMG patients were included. The mean age was 57.7 years, with a mean MG-ADL score of 10.86 ± 3.32. At week 4, MG-ADL scores showed a mean reduction of 6 points, reaching a maximum decline of 13 points. Among the patients, 85.7% (12/14) achieved MG-ADL responder status after one cycle of treatment. The most significant reduction in quantitative MG (QMG) scores also occurred at week 4, with a mean decrease of 7 points. Notably, the improvements in MG-ADL and QMG scores persisted until week 8. During treatment and follow-up period, only two mild neck rashes occurred and resolved promptly. No infections or SAE were reported. Discussion: A single cycle of efgartigimod treatment demonstrates effectiveness and the tolerability through week 8, with no new safety signals observed in Chinese gMG patients.
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