Batoclimab vs Placebo for Generalized Myasthenia Gravis

作者：Chong Yan, Yao-Xian Yue, Yuzhou Guan, Bitao Bu, Qing Ke, Rui‐Sheng Duan, Hui Deng, Qun Xue, Haishan Jiang, Fei Xiao, Huan Yang, Ting Chang, Zhang‐Yu Zou, Haifeng Li, Song Tan, Haibing Xiao, Hongyu Zhou, Hua Zhang, Qiang Meng, Wenyu Li, Wei Li, Junhong Guo, Yali Zhang, Zunbo Li, Jianglong Tu, Jian‐Quan Shi, Wěi Li, Michael Lee, Yu Chen, Xiaolu Tao, Shuai Zhao, Ping Li, Chongbo Zhao, Chongbo Zhao, Batoclimab Study Team, Jianying Xi, Chuanzhu Yan, Bin Zhang, Min Song, Zheng Rui, Xiaojun Ding, Cuiping Zhao, Cuiping Zhao, Ying Tan, Jiayu Shi, Jianwen Wang, Xiaoli Li, Bing Yang, Min Zhang, Congcong Wang, Xu Wang, Xingyi Xiao, Xiaopei Ji, Hui Zheng, Jing Luo, Hao Zhou, Huanhuan Li, Zhe Ruan, Lidong Jiao, Hui Lü, Jialin Chen, Sheng Chen, Hongbin Sun, Quantao Zeng, Yan Xie, Kai Chen, Li Zeng, Wenshuang Zeng, Qin Du, Jian Yin, Shifang Hou, Lei Zhang, Mingming Zhao, Ruihan Yang, Wenjiao Huang, Xingyue Hu, Luya Jin, Yuying Zhao, Tingjun Dai, Wei Zhang, Xueli Chang, Xue Bai, Xiuyun Li, Jiaojiao Ma, Ting Xiong, Jiaojiao Ma, Hong-Dong Zhao, Meng Zhang, Jun Dong · 发表于：JAMA Neurology · 年份：2024 · DOI：10.1001/jamaneurol.2024.0044 · 被引用次数：46 · 研究领域：Myasthenia Gravis and Thymoma、Adrenal Hormones and Disorders、Peripheral Neuropathies and Disorders

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 Participants: 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. 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 random...