"A multicenter, randomized, open-label, phase 2 clinical study of telitacicept in adult patients with generalized myasthenia gravis"

作者全名："Yin, Jian; Zhao, Mingming; Xu, Xianhao; Zhang, Meini; Xu, Zucai; Li, Zunbo; Qin, Xinyue; Li, Zhuyi; Zhao, Chongbo; Zhou, Hongyu; Ma, Ying; Cao, Wenfeng; Wang, Guoping; Lin, Yongzhong; Zhang, Jizhong; Zhang, Xu; Cai, Hongbin; Qian, Weidong; Wang, Yiqi; Zhang, Xinghu; Liu, Guangzhi; Wang, Jiawei; Qiu, Wei; Min, Lianqiu; Li, Jing; Deng, Hui; Chu, Lan; Zhang, Yifan; Fang, Jianmin"

作者地址："[Yin, Jian; Zhao, Mingming; Xu, Xianhao] Chinese Acad Med Sci, Beijing Hosp, Natl Ctr Gerontol, Dept Neurol,Inst Geriatr Med, Beijing 100730, Peoples R China; [Zhang, Meini] Shanxi Med Univ, Hosp 1, Dept Neurol, Taiyuan, Peoples R China; [Xu, Zucai] Zunyi Med Univ, Affiliated Hosp, Dept Neurol, Zunyi, Peoples R China; [Li, Zunbo] Xian Gaoxin Hosp, Dept Neurol, Xian, Peoples R China; [Qin, Xinyue] Chongqing Med Univ, Affiliated Hosp 1, Dept Neurol, Chongqing, Peoples R China; [Li, Zhuyi] Air Force Med Univ PLA, Tangdu Hosp, Dept Neurol, Xian, Peoples R China; [Zhao, Chongbo] Fudan Univ, Huashan Hosp, Dept Neurol, Shanghai, Peoples R China; [Zhou, Hongyu] Sichuan Univ, West China Hosp, Dept Neurol, Chengdu, Peoples R China; [Ma, Ying] North Sichuan Med Coll, Affiliated Hosp, Dept Neurol, Nanchong, Peoples R China; [Cao, Wenfeng] Jiangxi Prov Peoples Hosp, Dept Neurol, Nanchang, Peoples R China; [Wang, Guoping] Anhui Prov Hosp, Dept Neurol, Hefei, Peoples R China; [Lin, Yongzhong] Dalian Med Univ, Affiliated Hosp 2, Dept Neurol, Dalian, Peoples R China; [Zhang, Jizhong] Nanyang First Peoples Hosp, Dept Neurol, Nanyang 473000, Peoples R China; [Zhang, Xu] Wenzhou Med Univ, Affiliated Hosp 1, Dept Neurol, Wenzhou, Peoples R China; [Cai, Hongbin] Lanzhou Univ Second Hosp, Dept Neurol, Lanzhou, Peoples R China; [Qian, Weidong] Bengbu Med Coll, Affiliated Hosp 1, Dept Neurol, Bengbu, Peoples R China; [Wang, Yiqi] Zhejiang Prov Peoples Hosp, Dept Neurol, Hangzhou, Peoples R China; [Zhang, Xinghu] Capital Med Univ, Beijing Tiantan Hosp, Dept Neurol, Beijing, Peoples R China; [Liu, Guangzhi] Capital Med Univ, Beijing Anzhen Hosp, Dept Neurol, Beijing, Peoples R China; [Wang, Jiawei] Capital Med Univ, Beijing Tongren Hosp, Dept Neurol, Beijing, Peoples R China; [Qiu, Wei] Sun Yat Sen Univ, Affiliated Hosp 3, Dept Neurol, Guangzhou, Peoples R China; [Min, Lianqiu] Jinzhou Med Univ, Affiliated Hosp 1, Dept Neurol, Jinzhou, Peoples R China; [Li, Jing] Cent South Univ, Xiangya Hosp, Dept Neurol, Changsha, Peoples R China; [Deng, Hui] First Hosp Jilin Univ, Dept Neurol, Changchun, Peoples R China; [Chu, Lan; Zhang, Yifan] Guizhou Med Univ, Affiliated Hosp, Dept Neurol, Guiyang, Peoples R China; [Fang, Jianmin] Tongji Univ, Sch Life Sci & Technol, Shanghai 200092, Peoples R China"

通信作者："Yin, J (通讯作者)，Chinese Acad Med Sci, Beijing Hosp, Natl Ctr Gerontol, Dept Neurol,Inst Geriatr Med, Beijing 100730, Peoples R China.; Fang, JM (通讯作者)，Tongji Univ, Sch Life Sci & Technol, Shanghai 200092, Peoples R China."

来源：EUROPEAN JOURNAL OF NEUROLOGY

ESI学科分类：NEUROSCIENCE & BEHAVIOR

WOS号：WOS:001217192600001

JCR分区：Q1

影响因子：5.1

年份：2024

卷号：31

期号：8

开始页：　

结束页：　

文献类型：Article

关键词：autoimmune disease; B lymphocyte stimulator; clinical trial; immunotherapy; myasthenia gravis

摘要："Background and purposeThis study aimed to investigate the clinical efficacy and safety of telitacicept in patients with generalized myasthenia gravis (gMG) who tested positive for acetylcholine receptor antibodies or muscle-specific kinase antibodies and were receiving standard-of-care therapy.MethodsPatients meeting the eligibility criteria were randomly assigned to receive telitacicept subcutaneously once a week for 24 weeks in addition to standard-of-care treatment. The primary efficacy endpoint was the mean change in the quantitative myasthenia gravis (QMG) score from baseline to week 24. Secondary efficacy endpoints included mean change in QMG score from baseline to week 12 and gMG clinical absolute score from baseline to week 24. Additionally, safety, tolerability and pharmacodynamics were assessed.ResultsTwenty-nine of the 41 patients screened were randomly selected and enrolled. The mean (+/- standard deviation [SD]) reduction in QMG score from baseline to week 24 was 7.7 (+/- 5.34) and 9.6 (+/- 4.29) in the 160 mg and 240 mg groups, respectively. At week 12, mean reductions in QMG scores for these two groups were 5.8 (+/- 5.85) and 9.5 (+/- 5.03), respectively, indicating rapid clinical improvement. Safety analysis revealed no adverse events leading to discontinuation or mortalities. All patients showed consistent reductions in serum immunoglobulin (Ig) A, IgG and IgM levels throughout the study.ConclusionTelitacicept demonstrated safety, good tolerability and reduced clinical severity throughout the study period. Further validation of the clinical efficacy of telitacicept in gMG will be conducted in an upcoming phase 3 clinical trial."

基金机构：National High Level Hospital Clinical Reaearch Funding

基金资助正文：The authors thank all the patients who participated in our study. All the authors approved the submitted version for publication.