Observational studyFrontiers in immunology2024
Treatment of refractory immune-mediated necrotizing myopathy with efgartigimod.
Observational study in Frontiers in immunology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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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
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Who cites it
16 citing papers in PubMed.
- Emerging therapies in idiopathic inflammatory myopathies.Journal of neuromuscular diseases · 2026Review
- Targeting FcRn for immunomodulation: a promising therapy in autoimmune inflammatory rheumatic diseases.Inflammation research : official journal of the European Histamine Research Society ... [et al.] · 2026Review
- Autoantibody internalization in myositis skeletal muscle: Emerging evidence, mechanistic insights, and therapeutic relevance.Autoimmunity reviews · 2026Review
- Case Report: Dual response to efgartigimod in myasthenia gravis and comorbid autoimmune disorders: a case series.Frontiers in immunology · 2026Article
- A prospective real-world study of efgartigimod in the treatment of chronic inflammatory demyelinating polyradiculoneuropathy.Frontiers in immunology · 2026Article
- Autoimmune Neuromuscular Disorders at a Molecular Crossroad: Linking Pathogenesis to Targeted Immunotherapy.International journal of molecular sciences · 2025Review
- Efgartigimod for Generalized Myasthenia Gravis and Beyond: A Narrative Review of Its Pharmacological Profile, Clinical Utility, and Expanding Applications.Biomedicines · 2025Review
- The Effect of Anti-3-Hydroxy-3-Methylglutaryl-CoA Reductase (HMGCR) Human Autoantibodies on Muscle Regeneration in Mice.Muscle & nerve · 2025Article
- Targeted Therapy for Idiopathic Inflammatory Myopathy.Journal of cachexia, sarcopenia and muscle · 2025Review
- Juvenile dermatomyositis: new insights into pathogenesis and clinical applications.Pediatric rheumatology online journal · 2025Review
- Article
- Clinicopathological profiling and prognosis of idiopathic inflammatory myopathy overlapping with myasthenia gravis: a retrospective cohort study.Journal of neurology · 2025Article
- Advances in the Regulation by Immune Cells of Skeletal Myositis Outcomes.Aging and disease · 2025Review
- Local immunoglobulin expression in myositis is associated with interferon gamma signaling and correlates with disease activity.medRxiv : the preprint server for health sciences · 2025Article
- Targeting B Cells for the Treatment of Idiopathic Inflammatory Myopathy.Clinical reviews in allergy & immunology · 2025Review
- Efgartigimod combined with steroids as a fast-acting therapy for anti-SRP immune-mediated necrotizing myopathy.Frontiers in neurology · 2025Article
Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Objective: We aimed to explore the efficacy and safety of efgartigimod in patients with refractory immune-mediated necrotizing myopathy (IMNM). Methods: This open-label pilot observational study included seven patients with refractory IMNM, all of whom received intravenous efgartigimod treatment. The clinical response was assessed after 4 weeks of efgartigimod treatment according to the 2016 American College of Rheumatology-European League Against Rheumatism response criteria for adult idiopathic inflammatory myopathy. Serum levels of immunoglobulin as well as anti-signal recognition particle (SRP) and anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) antibodies were measured using enzyme-linked immunosorbent assays and commercial line immunoblot assays. Safety assessments included evaluations of adverse events and severe adverse events. Results: The seven patients with refractory IMNM included five cases with anti-HMGCR antibodies and two cases within anti-SRP antibodies. Four of the seven patients achieved clinical responses. The total improvement score for the responders at 4 weeks were 32.5, 40.0, 47.5, and 70.0, and those at 8 weeks were 27.5, 47.5, 57.5, and 70.0. In comparison to the responsive patients, the non-responsive patients had longer durations [8 (-) versus 2 (1-5) years, Conclusions: Despite its small size, our study demonstrated that promoting the degradation of endogenous immunoglobulin G may be effective for patients with IMNM. Efgartigimod may be a promising option for cases of refractory IMNM to shorten duration and minimize chronic myopathic features.
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