The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis
Abstract Background Myasthenia gravis (MG) is a chronic autoimmune disorder affecting the neuromuscular junction, where autoreactive immunoglobulin G (IgG) plays a key role in disease pathogenesis. The novel biologic Efgartigimod is a neonatal Fc receptor (FcRn) antagonist, promotes the lysosomal de...
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| Format: | Article |
| Language: | English |
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BMC
2025-08-01
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| Series: | European Journal of Medical Research |
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| Online Access: | https://doi.org/10.1186/s40001-025-03057-6 |
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| _version_ | 1849226525441785856 |
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| author | Jia-Jun Cheng Fu-Qiang Wang Zhang-Yi Dai Xin-Wei Wang Yun Wang |
| author_facet | Jia-Jun Cheng Fu-Qiang Wang Zhang-Yi Dai Xin-Wei Wang Yun Wang |
| author_sort | Jia-Jun Cheng |
| collection | DOAJ |
| description | Abstract Background Myasthenia gravis (MG) is a chronic autoimmune disorder affecting the neuromuscular junction, where autoreactive immunoglobulin G (IgG) plays a key role in disease pathogenesis. The novel biologic Efgartigimod is a neonatal Fc receptor (FcRn) antagonist, promotes the lysosomal degradation of IgG, and may offer a targeted approach for managing MG. Despite the growing interest in efgartigimod, there remains a lack of comprehensive evaluation of its efficacy and safety in different MG subtypes. Methods Comprehensive retrieval and screening were conducted on Pubmed, Embase, Web of Science, and Cochrane library to search studies on efgartigimod treatment. The data on response rates and adverse events were extracted, and the pooled effect size (ES) with the 95% confidence interval (CI) was calculated by fixed or random effect models. Sensitivity analysis and subgroup analysis were employed to test the heterogeneity. Funnel plots and trim-and-fill methods were used to test for publication bias. Results Data from 10 studies involving 305 patients were analyzed. The overall treatment response rate was 78% (95% CI: 67%–87%, I 2 = 73.4%). Subgroup analysis revealed pooled response rates of 79.2% (95% CI: 68.5%–88.4%, I 2 = 25.08%) in acetylcholine receptor antibody-positive MG (AChR+MG) patients and 76.2% (95% CI: 56.8%–91.5%, I 2 = 85.95%) in group that did not differentiate auto-antibody types. The pooled incidence of adverse events was 38% (95% CI: 17%–51%, I 2 = 92.59%), with infections (7%, 95% CI: 2%–14%, I 2 = 62.5%), headache (7%, 95% CI: 1%–18%, I 2 = 82.69%) and other (16%, 95% CI: 7%–28%, I 2 = 71.81%). Among them, grade 3–4 adverse events are 1% (95% CI: 0%–2%, I 2 = 0%). Conclusion Our study demonstrates that efgartigimod is highly effective and well-tolerated in patients with refractory MG. These findings suggest that efgartigimod is a promising drug for the treatment of MG. |
| format | Article |
| id | doaj-art-c15927c775bf4c5eacdc43b65ea1a672 |
| institution | Kabale University |
| issn | 2047-783X |
| language | English |
| publishDate | 2025-08-01 |
| publisher | BMC |
| record_format | Article |
| series | European Journal of Medical Research |
| spelling | doaj-art-c15927c775bf4c5eacdc43b65ea1a6722025-08-24T11:15:00ZengBMCEuropean Journal of Medical Research2047-783X2025-08-0130111210.1186/s40001-025-03057-6The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysisJia-Jun Cheng0Fu-Qiang Wang1Zhang-Yi Dai2Xin-Wei Wang3Yun Wang4Department of Thoracic Surgery, West China Hospital, Sichuan UniversityDepartment of Thoracic Surgery, West China Hospital, Sichuan UniversityDepartment of Thoracic Surgery, West China Hospital, Sichuan UniversitySchulich School of Medicine and Dentistry, Western UniversityDepartment of Thoracic Surgery, West China Hospital, Sichuan UniversityAbstract Background Myasthenia gravis (MG) is a chronic autoimmune disorder affecting the neuromuscular junction, where autoreactive immunoglobulin G (IgG) plays a key role in disease pathogenesis. The novel biologic Efgartigimod is a neonatal Fc receptor (FcRn) antagonist, promotes the lysosomal degradation of IgG, and may offer a targeted approach for managing MG. Despite the growing interest in efgartigimod, there remains a lack of comprehensive evaluation of its efficacy and safety in different MG subtypes. Methods Comprehensive retrieval and screening were conducted on Pubmed, Embase, Web of Science, and Cochrane library to search studies on efgartigimod treatment. The data on response rates and adverse events were extracted, and the pooled effect size (ES) with the 95% confidence interval (CI) was calculated by fixed or random effect models. Sensitivity analysis and subgroup analysis were employed to test the heterogeneity. Funnel plots and trim-and-fill methods were used to test for publication bias. Results Data from 10 studies involving 305 patients were analyzed. The overall treatment response rate was 78% (95% CI: 67%–87%, I 2 = 73.4%). Subgroup analysis revealed pooled response rates of 79.2% (95% CI: 68.5%–88.4%, I 2 = 25.08%) in acetylcholine receptor antibody-positive MG (AChR+MG) patients and 76.2% (95% CI: 56.8%–91.5%, I 2 = 85.95%) in group that did not differentiate auto-antibody types. The pooled incidence of adverse events was 38% (95% CI: 17%–51%, I 2 = 92.59%), with infections (7%, 95% CI: 2%–14%, I 2 = 62.5%), headache (7%, 95% CI: 1%–18%, I 2 = 82.69%) and other (16%, 95% CI: 7%–28%, I 2 = 71.81%). Among them, grade 3–4 adverse events are 1% (95% CI: 0%–2%, I 2 = 0%). Conclusion Our study demonstrates that efgartigimod is highly effective and well-tolerated in patients with refractory MG. These findings suggest that efgartigimod is a promising drug for the treatment of MG.https://doi.org/10.1186/s40001-025-03057-6EfgartigimodFcRnMyasthenia gravisMeta-analysisIgG |
| spellingShingle | Jia-Jun Cheng Fu-Qiang Wang Zhang-Yi Dai Xin-Wei Wang Yun Wang The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis European Journal of Medical Research Efgartigimod FcRn Myasthenia gravis Meta-analysis IgG |
| title | The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis |
| title_full | The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis |
| title_fullStr | The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis |
| title_full_unstemmed | The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis |
| title_short | The efficacy and safety of efgartigimod for refractory myasthenia gravis: a systematic review and meta-analysis |
| title_sort | efficacy and safety of efgartigimod for refractory myasthenia gravis a systematic review and meta analysis |
| topic | Efgartigimod FcRn Myasthenia gravis Meta-analysis IgG |
| url | https://doi.org/10.1186/s40001-025-03057-6 |
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