Efgartigimod for generalized myasthenia gravis: A multicenter real-world cohort study in China

作者全名："Luo, Sushan; Jiang, Qilong; Zeng, Wenshuang; Wang, Qinzhou; Zou, Zhangyu; Yu, Yanyan; Hong, Daojun; Zeng, Quantao; Tan, Song; Zhang, Zhouao; Zhang, Yong; Guo, Xiuming; Chen, Jing; Zhao, Zhongyan; Huang, Shixiong; Shi, Jianquan; Chen, Ying; Du, Lei; Yan, Chong; Xi, Jianying; Song, Jie; Zhao, Chongbo"

作者地址："[Luo, Sushan; Yan, Chong; Xi, Jianying; Song, Jie; Zhao, Chongbo] Fudan Univ, Shanghai Med Coll, Natl Ctr Neurol Disorders, Huashan Rare Dis Ctr,Huashan Hosp, Shanghai 200040, Peoples R China; [Luo, Sushan; Yan, Chong; Xi, Jianying; Song, Jie; Zhao, Chongbo] Fudan Univ, Huashan Hosp, Shanghai Med Coll, Natl Ctr Neurol Disorders,Dept Neurol, Shanghai 200040, Peoples R China; [Jiang, Qilong] Guangzhou Univ Chinese Med, Affiliated Hosp 1, Dept Myopathy, Guangzhou 510405, Peoples R China; [Zeng, Wenshuang] Hongkong Univ, Shenzhen Hosp, Dept Neurol, Shenzhen 518053, Peoples R China; [Wang, Qinzhou] Shandong Univ, Dept Neurol, Qilu Hosp, Jinan 250012, Peoples R China; [Zou, Zhangyu] Fujian Med Univ, Union Hosp, Dept Neurol, Fuzhou 350001, Peoples R China; [Yu, Yanyan; Hong, Daojun] Nanchang Univ, Affiliated Hosp 1, Dept Neurol, Nanchang 330006, Peoples R China; [Zeng, Quantao; Tan, Song] Univ Elect Sci & Technol China, Sichuan Prov Peoples Hosp, Dept Neurol, Chengdu 610072, Peoples R China; [Zhang, Zhouao; Zhang, Yong] Xuzhou Med Univ, Dept Neurol, Affiliated Hosp, Xuzhou 221004, Peoples R China; [Guo, Xiuming] Chongqing Med Univ, Affiliated Hosp 1, Dept Neurol, Chongqing 400016, Peoples R China; [Chen, Jing] Soochow Univ, Affiliated Hosp 2, Dept Neurol, Suzhou 215004, Peoples R China; [Zhao, Zhongyan; Huang, Shixiong] Hainan Med Univ, Hainan Gen Hosp, Hainan Affiliated Hosp, Dept Neurol, Haikou 570311, Peoples R China; [Shi, Jianquan] Nanjing Med Univ, Nanjing Hosp 1, Dept Neurol, Nanjing 210006, Peoples R China; [Chen, Ying] Wannan Med Coll, Affiliated Hosp 1, Dept Neurol, Wuhu 241004, Peoples R China; [Du, Lei] Xinjiang Med Univ, Affiliated Hosp 1, Dept Neurol, Urumqi 830054, Xinjiang Uygur, Peoples R China"

通信作者："Zhao, CB (通讯作者)，Fudan Univ, Huashan Hosp, Dept Neurol, Shanghai 200040, Peoples R China."

来源：ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY

ESI学科分类：NEUROSCIENCE & BEHAVIOR

WOS号：WOS:001263599100001

JCR分区：Q1

影响因子：5.3

年份：2024

卷号：11

期号：8

开始页：2212

结束页：2221

文献类型：Article

关键词：　

摘要："ObjectiveEfgartigimod, a neonatal Fc receptor antagonist, facilitates antibody degradation including pathogenic IgGs. The ADAPT study demonstrated the tolerability and efficacy of efgartigimod in the treatment of generalized myasthenia gravis (gMG). However, very limited evidence is available for the Chinese population, and it remains inconclusive about which kind of patients are selected to preferentially receive efgartigimod in real-world settings.MethodsThis multicenter cohort study included gMG patients treated at 14 neuromuscular reference centers in China. The Myasthenia Gravis Activities of Daily Living (MG-ADL) score, immunosuppressants, and the incidence of treatment-emergent adverse events (TEAEs) were prospectively collected.ResultsOf the 1640 gMG admitted between September and December 2023, 61 (3.7%) received efgartigimod for at least one treatment cycle. Among them, 56 cases (92%) were anti-AChR antibody-positive, 4 were anti-MuSK antibody-positive, and 1 was seronegative. Thymoma-associated myasthenia gravis accounted for most cases (44%, 27 out of 61). The principal causes of efgartigimod initiation included MG acute exacerbation (MGAE) (48%, 29 out of 61) and myasthenic crisis (MC) (15%, 9 out of 61). Clinically meaningful improvement was rapidly achieved in 97% (58 out of 61) of patients at 1.3 +/- 0.7 weeks. By week 12, the MG-ADL score reduced to 3.8 +/- 4.1 (baseline:10.5 +/- 5.2) for all participants, while it reduced to 4.0 +/- 4.7 for MGAE and 3.8 +/- 4.2 for MC, respectively. All but one TMG patient required no additional rescue therapies after efgartigimod initiation. 11.5% (7 out of 61) reported >= 1 TEAEs.InterpretationThis multicenter cohort study demonstrated the efficacy of efgartigimod in rapid control of gMG. Patients with MGAE or MC would benefit from efgartigimod treatment."

基金机构："National Key Research and Development Plan of China [2022YFC3501303, 2022YFC3501305]; National Natural Science Foundation of China [82071410, 82001335]"

基金资助正文：This work was supported by financial grants from the National Key Research and Development Plan of China (2022YFC3501303 and 2022YFC3501305) and the National Natural Science Foundation of China (82071410 and 82001335).