Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis
ObjectiveEfgartigimod (EFG), a neonatal Fc receptor antagonist that facilitates the degradation of pathogenic immunoglobulin G, is approved for the treatment of generalized myasthenia gravis (MG). This study aims to evaluate the efficacy and safety of EFG in patients with very-late-onset myasthenia...
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Frontiers Media S.A.
2025-04-01
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| Series: | Frontiers in Immunology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fimmu.2025.1579859/full |
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| author | Zhouao Zhang Mingjin Yang Mingjin Yang Mingjin Yang Xinyan Guo Xinyan Guo Tianyu Ma Tianyu Ma Zhouyi Wang Zhouyi Wang Zhouyi Wang Tiancheng Luo Tiancheng Luo Tiancheng Luo Deyou Peng Deyou Peng Xue Du Xue Du Xiaoyu Huang Yong Zhang |
| author_facet | Zhouao Zhang Mingjin Yang Mingjin Yang Mingjin Yang Xinyan Guo Xinyan Guo Tianyu Ma Tianyu Ma Zhouyi Wang Zhouyi Wang Zhouyi Wang Tiancheng Luo Tiancheng Luo Tiancheng Luo Deyou Peng Deyou Peng Xue Du Xue Du Xiaoyu Huang Yong Zhang |
| author_sort | Zhouao Zhang |
| collection | DOAJ |
| description | ObjectiveEfgartigimod (EFG), a neonatal Fc receptor antagonist that facilitates the degradation of pathogenic immunoglobulin G, is approved for the treatment of generalized myasthenia gravis (MG). This study aims to evaluate the efficacy and safety of EFG in patients with very-late-onset myasthenia gravis (VLOMG).MethodsThis study enrolled 15 consecutive patients diagnosed with VLOMG who received EFG treatment. Baseline demographic and clinical characteristics, as well as dynamic changes in the MG-specific activities of daily living (MG-ADL) score and quantitative MG (QMG) score, were systematically recorded.ResultsPatients were stratified into two groups: a worse group (n = 8) and a new-diagnosed group (n = 7), the latter of which included 5 patients who had received monotherapy with pyridostigmine (Py) prior to EFG. At week 5, the mean changes in MG-ADL scores were -4.9 ± 3.3 in the overall VLOMG cohort, -6.1 ± 3.1 in the new-diagnosed group, -6.6 ± 3.6 in the mono-Py subgroup, and -3.8 ± 3.2 in the worse group. The clinical meaningful improvement (CMI) rate was 86.7% (13/15) in the overall cohort, 75.0% (6/8) in the worse group, and 100.0% (7/7) in the new-diagnosed group. During a mean follow-up time of 39.2 ± 16.2 weeks, symptoms remained stable in responsive patients, with various treatment strategies implemented following the fast-acting treatment of EFG. No adverse drug reactions were reported in this cohort.ConclusionThis study demonstrates that EFG is an effective and safe treatment for patients with VLOMG. EFG exhibits potential as an early, fast-acting treatment and may confer sustained clinical benefits in this patient population. |
| format | Article |
| id | doaj-art-6d2f9cfaa9bd439385be406181c3f9a1 |
| institution | OA Journals |
| issn | 1664-3224 |
| language | English |
| publishDate | 2025-04-01 |
| publisher | Frontiers Media S.A. |
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| series | Frontiers in Immunology |
| spelling | doaj-art-6d2f9cfaa9bd439385be406181c3f9a12025-08-20T02:26:41ZengFrontiers Media S.A.Frontiers in Immunology1664-32242025-04-011610.3389/fimmu.2025.15798591579859Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravisZhouao Zhang0Mingjin Yang1Mingjin Yang2Mingjin Yang3Xinyan Guo4Xinyan Guo5Tianyu Ma6Tianyu Ma7Zhouyi Wang8Zhouyi Wang9Zhouyi Wang10Tiancheng Luo11Tiancheng Luo12Tiancheng Luo13Deyou Peng14Deyou Peng15Xue Du16Xue Du17Xiaoyu Huang18Yong Zhang19Department of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaFirst Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu, ChinaCentral Laboratory, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaFirst Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaFirst Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaFirst Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu, ChinaCentral Laboratory, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaFirst Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu, ChinaCentral Laboratory, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaFirst Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaFirst Clinical Medical College, Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaDepartment of Neurology, The Affiliated Hospital of Xuzhou Medical University, Xuzhou, Jiangsu, ChinaObjectiveEfgartigimod (EFG), a neonatal Fc receptor antagonist that facilitates the degradation of pathogenic immunoglobulin G, is approved for the treatment of generalized myasthenia gravis (MG). This study aims to evaluate the efficacy and safety of EFG in patients with very-late-onset myasthenia gravis (VLOMG).MethodsThis study enrolled 15 consecutive patients diagnosed with VLOMG who received EFG treatment. Baseline demographic and clinical characteristics, as well as dynamic changes in the MG-specific activities of daily living (MG-ADL) score and quantitative MG (QMG) score, were systematically recorded.ResultsPatients were stratified into two groups: a worse group (n = 8) and a new-diagnosed group (n = 7), the latter of which included 5 patients who had received monotherapy with pyridostigmine (Py) prior to EFG. At week 5, the mean changes in MG-ADL scores were -4.9 ± 3.3 in the overall VLOMG cohort, -6.1 ± 3.1 in the new-diagnosed group, -6.6 ± 3.6 in the mono-Py subgroup, and -3.8 ± 3.2 in the worse group. The clinical meaningful improvement (CMI) rate was 86.7% (13/15) in the overall cohort, 75.0% (6/8) in the worse group, and 100.0% (7/7) in the new-diagnosed group. During a mean follow-up time of 39.2 ± 16.2 weeks, symptoms remained stable in responsive patients, with various treatment strategies implemented following the fast-acting treatment of EFG. No adverse drug reactions were reported in this cohort.ConclusionThis study demonstrates that EFG is an effective and safe treatment for patients with VLOMG. EFG exhibits potential as an early, fast-acting treatment and may confer sustained clinical benefits in this patient population.https://www.frontiersin.org/articles/10.3389/fimmu.2025.1579859/fullvery-late-onset myasthenia gravisfast-acting treatmentefgartigimodMG-ADLclinical meaningful improvement |
| spellingShingle | Zhouao Zhang Mingjin Yang Mingjin Yang Mingjin Yang Xinyan Guo Xinyan Guo Tianyu Ma Tianyu Ma Zhouyi Wang Zhouyi Wang Zhouyi Wang Tiancheng Luo Tiancheng Luo Tiancheng Luo Deyou Peng Deyou Peng Xue Du Xue Du Xiaoyu Huang Yong Zhang Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis Frontiers in Immunology very-late-onset myasthenia gravis fast-acting treatment efgartigimod MG-ADL clinical meaningful improvement |
| title | Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis |
| title_full | Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis |
| title_fullStr | Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis |
| title_full_unstemmed | Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis |
| title_short | Efgartigimod as a fast-acting treatment in generalized very-late-onset myasthenia gravis |
| title_sort | efgartigimod as a fast acting treatment in generalized very late onset myasthenia gravis |
| topic | very-late-onset myasthenia gravis fast-acting treatment efgartigimod MG-ADL clinical meaningful improvement |
| url | https://www.frontiersin.org/articles/10.3389/fimmu.2025.1579859/full |
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