艾加莫德治疗重症肌无力的现状与未来展望：从机制到真实世界实践
Current Status and Future Prospects of Efgartigimod in the Treatment of Myasthenia Gravis: From Mechanism to Real-World Practice
DOI: 10.12677/acm.2025.15123671, PDF,   
作者: 牟 阳, 余 刚*：重庆医科大学第一附属医院神经内科，重庆
关键词: 重症肌无力；艾加莫德；真实世界应用；Myasthenia Gravis； Efgartigimod； Real-World Applications
摘要: 重症肌无力(MG)是一种由自身抗体介导的神经肌肉接头传递障碍性疾病，表现为波动性骨骼肌无力，严重影响患者生活质量。传统免疫治疗虽广泛使用，但部分患者仍面临疗效不足、激素依赖及严重不良反应等问题。近年来，靶向治疗的突破为MG管理提供了新方向。其中艾加莫德作为首个新生儿Fc受体(FcRn)拮抗剂，通过降低致病性抗体免疫球蛋白G (IgG)水平，成为治疗乙酰胆碱受体(AChR)抗体阳性的全身型MG (gMG)的重要选择。本文系统综述艾加莫德的作用机制、临床疗效、安全性以及在真实世界中的应用场景，探讨其在MG治疗中的价值，为艾加莫德的临床应用提供基于真实世界证据的决策支持。
Abstract: Myasthenia gravis (MG) is an autoimmune disorder mediated by autoantibodies that impair neuromuscular transmission, resulting in fluctuating muscle weakness and diminished quality of life. Although conventional therapies are widely used, they remain limited by inadequate responses, corticosteroid dependence, and significant adverse effects. Recent advances in targeted therapies have introduced new treatment options. Efgartigimod, the first neonatal Fc receptor (FcRn) antagonist, lowers pathogenic IgG levels and represents an important therapeutic option for acetylcholine receptor (AChR) antibody–positive generalized MG (gMG). This review summarizes the mechanism of action, efficacy, and safety profile of efgartigimod, with particular emphasis on real-world applications. We further evaluate its role in MG management and provide real-world evidence to inform clinical decision-making.
文章引用：牟阳, 余刚. 艾加莫德治疗重症肌无力的现状与未来展望：从机制到真实世界实践[J]. 临床医学进展, 2025, 15(12): 2412-2418. https://doi.org/10.12677/acm.2025.15123671

参考文献

[1] Jiang, S., Li, H., Zhang, L., Mu, W., Zhang, Y., Chen, T., et al. (2024) Generic Diagramming Platform (GDP): A Comprehensive Database of High-Quality Biomedical Graphics. Nucleic Acids Research, 53, D1670-D1676. [Google Scholar] [CrossRef] [PubMed]
[2] Ulrichts, P., Guglietta, A., Dreier, T., van Bragt, T., Hanssens, V., Hofman, E., et al. (2018) Neonatal Fc Receptor Antagonist Efgartigimod Safely and Sustainably Reduces Iggs in Humans. Journal of Clinical Investigation, 128, 4372-4386. [Google Scholar] [CrossRef] [PubMed]
[3] Howard Jr., J.F., Bril, V., Burns, T.M., et al. (2019) Randomized Phase 2 Study of FcRn Antagonist Efgartigimod in Generalized Myasthenia Gravis. Neurology, 92, e2661-e2673.
[4] Howard, J.F., Bril, V., Vu, T., Karam, C., Peric, S., Margania, T., et al. (2021) Safety, Efficacy, and Tolerability of Efgartigimod in Patients with Generalised Myasthenia Gravis (ADAPT): A Multicentre, Randomised, Placebo-Controlled, Phase 3 Trial. The Lancet Neurology, 20, 526-536. [Google Scholar] [CrossRef] [PubMed]
[5] Habib, A.A., Claeys, K.G., Bril, V., Hussain, Y., Gwathmey, K., Sahagian, G., et al. (2025) ADAPT NXT: Fixed Cycles or Every‐Other‐Week IV Efgartigimod in Generalized Myasthenia Gravis. Annals of Clinical and Translational Neurology, 12, 1162-1170. [Google Scholar] [CrossRef] [PubMed]
[6] Howard, J.F., Vu, T., Li, G., Korobko, D., Smilowski, M., Liu, L., et al. (2024) Subcutaneous Efgartigimod PH20 in Generalized Myasthenia Gravis: A Phase 3 Randomized Noninferiority Study (ADAPT-SC) and Interim Analyses of a Long-Term Open-Label Extension Study (ADAPT-SC+). Neurotherapeutics, 21, e00378. [Google Scholar] [CrossRef] [PubMed]
[7] Katyal, N., Halldorsdottir, K., Govindarajan, R., Shieh, P., Muley, S., Reyes, P., et al. (2023) Safety and Outcomes with Efgartigimod Use for Acetylcholine Receptor‐positive Generalized Myasthenia Gravis in Clinical Practice. Muscle & Nerve, 68, 762-766. [Google Scholar] [CrossRef] [PubMed]
[8] Luo, S., Jiang, Q., Zeng, W., Wang, Q., Zou, Z., Yu, Y., et al. (2024) Efgartigimod for Generalized Myasthenia Gravis: A Multicenter Real‐World Cohort Study in China. Annals of Clinical and Translational Neurology, 11, 2212-2221. [Google Scholar] [CrossRef] [PubMed]
[9] Remijn‐Nelissen, L., Tannemaat, M.R., Ruiter, A.M., Campman, Y.J.M. and Verschuuren, J.J.G.M. (2024) Efgartigimod in Refractory Autoimmune Myasthenia Gravis. Muscle & Nerve, 70, 325-332. [Google Scholar] [CrossRef] [PubMed]
[10] Dewilde, S., Griffiths, A., Qi, C.Z., Phillips, G., Gelinas, D., Brauer, E., et al. (2024) Post-Hoc Analyses from the ADAPT Clinical Study Demonstrate Aggregate Sustained Benefit of Efgartigimod in Generalized Myasthenia Gravis. Journal of the Neurological Sciences, 466, Article ID: 123264. [Google Scholar] [CrossRef] [PubMed]
[11] Wang, P., Zhang, B., Yin, J., Xi, J., Tan, Y., Gao, F., et al. (2024) Prospective Cohort Study Evaluating Efficacy and Safety of Efgartigimod in Chinese Generalized Myasthenia Gravis Patients. Frontiers in Neurology, 15, Article 1407418. [Google Scholar] [CrossRef] [PubMed]
[12] Horiuchi, K., Nakamura, S., Yamada, K., Inoue, T. and Oiwa, K. (2024) Retrospective Analysis of Efgartigimod Use in Patients with Double-Seronegative Generalized Myasthenia Gravis: A Case Series. Neuromuscular Disorders, 39, 37-41. [Google Scholar] [CrossRef] [PubMed]
[13] Sorrenti, B., Laurini, C., Bosco, L., Strano, C.M.M., Scarlato, M., Gastaldi, M., et al. (2024) Overcoming Therapeutic Challenges: Successful Management of a Supposedly Triple Seronegative, Refractory Generalized Myasthenia Gravis Patient with Efgartigimod. European Journal of Neurology, 31, e16306. [Google Scholar] [CrossRef] [PubMed]
[14] Usmani, A., Kwan, L., Wahib‐Khalil, D., Trivedi, J., Nations, S. and Sarode, R. (2019) Excellent Response to Therapeutic Plasma Exchange in Myasthenia Gravis Patients Irrespective of Antibody Status. Journal of Clinical Apheresis, 34, 416-422. [Google Scholar] [CrossRef] [PubMed]
[15] Dos Santos, J.B.R., Gomes, R.M. and da Silva, M.R.R. (2022) Abdeg Technology for the Treatment of Myasthenia Gravis: Efgartigimod Drug Experience. Expert Review of Clinical Immunology, 18, 879-888. [Google Scholar] [CrossRef] [PubMed]
[16] Zhu, G., Ma, Y., Zhou, H., Nie, X., Qi, W., Hao, L., et al. (2024) Case Report: Rapid Clinical Improvement in Acute Exacerbation of Musk-MG with Efgartigimod. Frontiers in Immunology, 15, Article 1401972. [Google Scholar] [CrossRef] [PubMed]
[17] Shi, F., Chen, J., Feng, L., Lai, R., Zhou, H., Sun, X., et al. (2024) Efgartigimod Treatment in Patients with Anti-Musk-Positive Myasthenia Gravis in Exacerbation. Frontiers in Neurology, 15, Article 1486659. [Google Scholar] [CrossRef] [PubMed]
[18] Bril, V., Drużdż, A., Grosskreutz, J., Habib, A.A., Mantegazza, R., Sacconi, S., et al. (2023) Safety and Efficacy of Rozanolixizumab in Patients with Generalised Myasthenia Gravis (MycarinG): A Randomised, Double-Blind, Placebo-Controlled, Adaptive Phase 3 Study. The Lancet Neurology, 22, 383-394. [Google Scholar] [CrossRef] [PubMed]
[19] Antozzi, C., Guptill, J., Bril, V., Gamez, J., Meuth, S.G., Nowak, R.J., et al. (2024) Safety and Efficacy of Nipocalimab in Patients with Generalized Myasthenia Gravis: Results from the Randomized Phase 2 Vivacity-MG Study. Neurology, 102, e207937. [Google Scholar] [CrossRef] [PubMed]
[20] Zhang, Z., Yang, M., Luo, T., Du, X., Wang, Z., Huang, X., et al. (2024) Rescue Treatment with Add-On Efgartigimod in a Patient with Impending Myasthenic Crisis: A Case Report. Therapeutic Advances in Neurological Disorders, 17, 1-6. [Google Scholar] [CrossRef] [PubMed]
[21] Kawama, K., Warabi, Y., Bokuda, K., Kimura, H. and Takahashi, K. (2023) Exacerbation of Thymoma-Associated Myasthenia Gravis Following Efgartigimod Treatment Related to Anti-Acetylcholine Receptor Antibody Overshoot: A Report of Two Cases. Cureus, 15, e50692. [Google Scholar] [CrossRef] [PubMed]
[22] Wang, S., Wang, Q., Jin, L., Dong, J. and Ding, J. (2024) Efgartigimod Is a New Option for the Treatment of Thymoma Associated Myasthenia Gravis: A Case Report. International Journal of Surgery Case Reports, 115, Article ID: 109241. [Google Scholar] [CrossRef] [PubMed]
[23] Gilhus, N.E. (2023) Treatment Considerations in Myasthenia Gravis for the Pregnant Patient. Expert Review of Neurotherapeutics, 23, 169-177. [Google Scholar] [CrossRef] [PubMed]
[24] Li, Y., Dong, X., Li, Z., Peng, Y., Jin, W., Zhou, R., et al. (2021) Characteristics of Myasthenia Gravis in Elderly Patients: A Retrospective Study. Neurological Sciences, 43, 2775-2783. [Google Scholar] [CrossRef] [PubMed]
[25] Inzirillo, K., Carranza, O. and Swerdloff, M.A. (2024) Intravenous Efgartigimod Alfa as Initial Monotherapy for Disabling Ocular Myasthenia in an Elderly Patient with Multiple Comorbidities. Cureus, 16, e74768. [Google Scholar] [CrossRef] [PubMed]
[26] 中华医学会神经病学分会神经免疫学组. 中国重症肌无力诊断和治疗指南(2025版) [J]. 中华神经科杂志, 2025, 58(7): 721-741.
[27] Magro, D., Venezia, M. and Rita Balistreri, C. (2024) The Omics Technologies and Liquid Biopsies: Advantages, Limitations, Applications. Medicine in Omics, 11, Article ID: 100039. [Google Scholar] [CrossRef] 
[28] Terriaca, S., Ferlosio, A., Scioli, M.G., Coppa, F., Bertoldo, F., Pisano, C., et al. (2024) Mirna Regulation of Cell Phenotype and Parietal Remodeling in Atherosclerotic and Non-Atherosclerotic Aortic Aneurysms: Differences and Similarities. International Journal of Molecular Sciences, 25, Article 2641. [Google Scholar] [CrossRef] [PubMed]
[29] Balistreri, C.R. (2022) Promising Strategies for Preserving Adult Endothelium Health and Reversing Its Dysfunction: From Liquid Biopsy to New Omics Technologies and Noninvasive Circulating Biomarkers. International Journal of Molecular Sciences, 23, Article 7548. [Google Scholar] [CrossRef] [PubMed]
[30] Liu, P., Jiang, Y., Gu, S., Xue, Y., Yang, H., Li, Y., et al. (2021) Metagenome-Wide Association Study of Gut Microbiome Revealed Potential Microbial Marker Set for Diagnosis of Pediatric Myasthenia Gravis. BMC Medicine, 19, Article No. 159. [Google Scholar] [CrossRef] [PubMed]
[31] Sikorski, P., Li, Y., Cheema, M., Wolfe, G.I., Kusner, L.L., Aban, I., et al. (2023) Serum Metabolomics of Treatment Response in Myasthenia Gravis. PLOS ONE, 18, e0287654. [Google Scholar] [CrossRef] [PubMed]
[32] Blackmore, D., Siddiqi, Z., Li, L., Wang, N. and Maksymowych, W. (2019) Beyond the Antibodies: Serum Metabolomic Profiling of Myasthenia Gravis. Metabolomics, 15, Article No. 109. [Google Scholar] [CrossRef] [PubMed]

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