机构：[1]National Center for Neurological Disorders, Huashan Rare Disease Centre, Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China[2]Department of Neurology, Qilu Hospital (Qingdao), Shandong University, Qingdao, China[3]Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing, China[4]Department of Neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,Wuhan, China华中科技大学同济医学院附属同济医院[5]Department of Neurology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China浙江大学医学院附属第一医院[6]Department of Neurology, the First Affiliated Hospital of Shandong First Medical University & Shandong Provincial Qianfoshan Hospital, Ji’nan, China[7]Department of Neurology and Neuroscience Center, the First Hospital of Jilin University, Changchun, China[8]Department of Neurology, the First Affiliated Hospital of Soochow University, Suzhou, China[9]Department of Neurology, Nanfang Hospital, Southern Medical University, Guangzhou, China[10]Department of Neurology, the First Affiliated Hospital of Chongqing Medical University, Chongqing, China重庆医科大学附属第一医院[11]Department of Neurology, Xiangya Hospital, Central South University, Changsha, China[12]Department of Neurology, Tangdu Hospital, the Fourth Military Medical University, Xi’an, China[13]Department of Neurology, Fujian Medical University Union Hospital, Fuzhou, China[14]Department of Neurology, Xuanwu Hospital, Capital Medical University, Beijing, China首都医科大学宣武医院[15]Department of Neurology, Sichuan Provincial People’s Hospital, School of Medicine, University of Electronic Science and Technology of China, Chengdu, China四川省人民医院[16]Neuromedicine Center, Department of Neurology, The University of Hong Kong—Shenzhen Hospital, Shenzhen, China深圳市康宁医院深圳医学信息中心香港大学深圳医院[17]Department of Neurology,West China Hospital, Sichuan University, Chengdu, China四川大学华西医院[18]Department of Neurology, Beijing Hospital, Beijing, China[19]Department of Neurology, the First People’s Hospital of Yunnan Province & the Affiliated Hospital of Kunming University of Science and Technology, Kunming, China内科片神经内科云南省第一人民医院[20]Department of Neurology, Sir Run Run Shaw Hospital, School of Medicine, Zhejiang University, Hangzhou, China[21]Department of Neurology, Qilu Hospital, Shandong University, Ji’nan, China[22]Department of Neurology, First Hospital, Shanxi Medical University, Taiyuan, China[23]Department of Neurology, Chifeng Municipal Hospital, Chifeng, China[24]Department of Neurology, Xi’an Gaoxin Hospital, Xi’an Medical College, Xi’an, China[25]Department of Neurology, the Second Affiliated Hospital of Nanchang University, Nanchang, China[26]Department of Neurology, Nanjing First Hospital, Nanjing Medical University, Nanjing, China[27]Department of Neurology, Army Medical Center of PLA, Army Medical University, Chongqing, China[28]Nona Biosciences (Suzhou) Co Ltd, Shanghai, China
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.To examine the efficacy and safety profile of batoclimab, a monoclonal IgG1 antibody, in patients with generalized MG.This 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.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.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.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.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.ClinicalTrials.gov Identifier: NCT05039190.
基金：
This trial was funded by Nona
Biosciences (Suzhou).
第一作者机构：[1]National Center for Neurological Disorders, Huashan Rare Disease Centre, Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China
共同第一作者：
通讯作者：
通讯机构：[1]National Center for Neurological Disorders, Huashan Rare Disease Centre, Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China[*1]National Center for Neurological Disorders, Huashan Rare Disease Centre, Department of Neurology, Huashan Hospital, Fudan University, No.12Wulumuqi Middle Road, Shanghai 200040, China
推荐引用方式(GB/T 7714)：
Yan Chong,Yue Yaoxian,Guan Yuzhou,et al.Batoclimab vs Placebo for Generalized Myasthenia Gravis: A Randomized Clinical Trial[J].JAMA NEUROLOGY.2024,81(4):336-345.doi:10.1001/jamaneurol.2024.0044.
APA：
Yan Chong,Yue Yaoxian,Guan Yuzhou,Bu Bitao,Ke Qing...&Zhao Chongbo.(2024).Batoclimab vs Placebo for Generalized Myasthenia Gravis: A Randomized Clinical Trial.JAMA NEUROLOGY,81,(4)
MLA：
Yan Chong,et al."Batoclimab vs Placebo for Generalized Myasthenia Gravis: A Randomized Clinical Trial".JAMA NEUROLOGY 81..4(2024):336-345
医学