Batoclimab vs Placebo for Generalized Myasthenia Gravis

作者全名："Yan, Chong; Yue, Yaoxian; Guan, Yuzhou; Bu, Bitao; Ke, Qing; Duan, Ruisheng; Deng, Hui; Xue, Qun; Jiang, Haishan; Xiao, Fei; Yang, Huan; Chang, Ting; Zou, Zhangyu; Li, Haifeng; Tan, Song; Xiao, Haibing; Zhou, Hongyu; Zhang, Hua; Meng, Qiang; Li, Wenyu; Li, Wei; Guo, Junhong; Zhang, Yali; Li, Zunbo; Tu, Jianglong; Shi, Jianquan; Li, Wei; Lee, Michael; Chen, Yu; Tao, Xiaolu; Zhao, Shuai; Li, Ping; Zhao, Chongbo"

作者地址："[Yan, Chong; Zhao, Chongbo] Fudan Univ, Huashan Hosp, Natl Ctr Neurol Disorders, Huashan Rare Dis Ctr,Dept Neurol, Shanghai, Peoples R China; [Yue, Yaoxian] Shandong Univ, Qilu Hosp Qingdao, Dept Neurol, Qingdao, Peoples R China; [Guan, Yuzhou] Peking Union Med Coll Hosp, Chinese Acad Med Sci, Dept Neurol, Beijing, Peoples R China; [Bu, Bitao] Huazhong Univ Sci & Technol, Tongji Hosp, Tongji Med Coll, Dept Neurol, Wuhan, Peoples R China; [Ke, Qing] Zhejiang Univ, Sch Med, Affiliated Hosp 1, Dept Neurol, Hangzhou, Peoples R China; [Duan, Ruisheng] Shandong First Med Univ, Dept Neurol, Affiliated Hosp 1, Jinan, Peoples R China; [Duan, Ruisheng] Shandong Prov Qianfoshan Hosp, Jinan, Peoples R China; [Deng, Hui] Jilin Univ, Neurosci Ctr, Dept Neurol, Hosp 1, Changchun, Peoples R China; [Xue, Qun] Soochow Univ, Dept Neurol, Affiliated Hosp 1, Suzhou, Peoples R China; [Jiang, Haishan] Southern Med Univ, Nanfang Hosp, Dept Neurol, Guangzhou, Peoples R China; [Xiao, Fei] Chongqing Med Univ, Dept Neurol, Affiliated Hosp 1, Chongqing, Peoples R China; [Yang, Huan] Cent South Univ, Xiangya Hosp, Dept Neurol, Changsha, Peoples R China; [Chang, Ting] Fourth Mil Med Univ, Tangdu Hosp, Dept Neurol, Xian, Peoples R China; [Zou, Zhangyu] Fujian Med Univ, Union Hosp, Dept Neurol, Fuzhou, Peoples R China; [Li, Haifeng] Capital Med Univ, Xuanwu Hosp, Dept Neurol, Beijing, Peoples R China; [Tan, Song] Univ Elect Sci & Technol China, Sichuan Prov Peoples Hosp, Sch Med, Dept Neurol, Chengdu, Peoples R China; [Xiao, Haibing] Univ Hong Kong, Shenzhen Hosp, Neuromed Ctr, Dept Neurol, Shenzhen, Peoples R China; [Zhou, Hongyu] Sichuan Univ, West China Hosp, Dept Neurol, Chengdu, Peoples R China; [Zhang, Hua] Beijing Hosp, Dept Neurol, Beijing, Peoples R China; [Meng, Qiang] Kunming Univ Sci & Technol, Peoples Hosp Yunnan Prov 1, Dept Neurol, Kunming, Peoples R China; [Meng, Qiang; Li, Wei] Kunming Univ Sci & Technol, Affiliated Hosp, Kunming, Peoples R China; [Li, Wenyu] Zhejiang Univ, Sir Run Run Shaw Hosp, Sch Med, Dept Neurol, Hangzhou, Peoples R China; Shandong Univ, Qilu Hosp, Dept Neurol, Jinan, Peoples R China; [Guo, Junhong] Shanxi Med Univ, Hosp 1, Dept Neurol, Taiyuan, Peoples R China; [Zhang, Yali] Chifeng Municipal Hosp, Dept Neurol, Chifeng, Peoples R China; [Li, Zunbo] Xian Gaoxin Hosp, Dept Neurol, Xian Med Coll, Xian, Peoples R China; [Tu, Jianglong; Li, Wei] Nanchang Univ, Dept Neurol, Affiliated Hosp 2, Nanchang, Jiangxi, Peoples R China; [Shi, Jianquan] Nanjing Med Univ, Nanjing Hosp 1, Dept Neurol, Nanjing, Peoples R China; Army Med Univ, Army Med Ctr PLA, Dept Neurol, Chongqing, Peoples R China; [Lee, Michael; Chen, Yu; Tao, Xiaolu; Zhao, Shuai; Li, Ping] Nona Biosci Suzhou Co Ltd, Shanghai, Peoples R China"

通信作者："Zhao, CB (通讯作者)，Fudan Univ, Huashan Hosp, Natl Ctr Neurol Disorders, Huashan Rare Dis Ctr,Dept Neurol, Shanghai, Peoples R China."

来源：JAMA NEUROLOGY

ESI学科分类：NEUROSCIENCE & BEHAVIOR

WOS号：WOS:001181352000003

JCR分区：Q1

影响因子：20.4

年份：2024

卷号：　

期号：　

开始页：　

结束页：　

文献类型：Article; Early Access

关键词：　

摘要："Importance Myasthenia gravis (MG) is caused by autoantibodies that disrupt the neuromuscular junction. The neonatal fragment crystallizable receptor (FcRn) antagonists, efgartigimod and rozanolixizumab, reduce immunoglobulin G (IgG) level in the circulation and alleviate symptoms in patients with generalized MG. Objective To examine the efficacy and safety profile of batoclimab, a monoclonal IgG1 antibody, in patients with generalized MG. Design, Setting, and ParticipantsThis was a multicenter randomized clinical trial conducted from September 15, 2021, to June 29, 2022, at 27 centers in China. Adult patients 18 years or older with generalized MG were screened, and those who were antibody positive were enrolled. Intervention Eligible patients received batoclimab or matching placebo in addition to standard of care. Each treatment cycle consisted of 6 weekly subcutaneous injections of batoclimab, 680 mg, or matching placebo followed by 4 weeks of observation. A second treatment cycle was conducted in patients who required continuing treatment. Main Outcome and Measure The primary outcome was sustained improvement, as defined by a 3-point or greater reduction in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score from baseline for 4 or more consecutive weeks in the first cycle in individuals who were positive for acetylcholine receptor or muscle-specific kinase antibodies. Results A total of 178 adult patients with generalized MG were screened, 132 were randomly assigned, 131 tested positive for antibodies, and 1 tested negative for antibodies. A total of 132 patients (mean [SE] age, 43.8 [13.6] years; 88 women [67.2%]) were enrolled. The rate of sustained MG-ADL improvement in the first cycle in antibody-positive patients was 31.3% (20 of 64) in the placebo group vs 58.2% (39 of 67) in the batoclimab group (odds ratio, 3.45; 95% CI, 1.62-7.35; P = .001). The MG-ADL score diverged between the 2 groups as early as week 2. The mean (SE) maximum difference in MG-ADL score reduction occurred 1 week after the last dose (day 43, 1.7 [0.3] in the placebo group vs 3.6 [0.3] in the batoclimab group; group difference, -1.9; 95% CI, -2.8 to -1.0; nominal P < .001). The rates of treatment-related and severe treatment-emergent adverse events in patients were 36.9% (24 of 65) and 7.7% (5 of 65) in the placebo group vs 70.1% (47 of 67) and 3.0% (2 of 67) in the batoclimab group, respectively. Conclusions and Relevance Batoclimab increased the rate of sustained MG-ADL improvement and was well tolerated in adult patients with generalized MG. Clinical effects and the extent of IgG reduction were similar to those previously reported for efgartigimod and rozanolixizumab. Future studies of large sample size are needed to further understand the safety profile of batoclimab."

基金机构：Nona Biosciences (Suzhou)

基金资助正文：No Statement Available