Tocilizumab Monotherapy or Combined With Methotrexate for Rheumatoid Arthritis

作者：Tian Liu, Li Wang, Xiao Zhang, Linjie Chen, Yi Liu, Zhenyu Jiang, Zongwen Shuai, Miaojia Zhang, Wei Wei, Huaxiang Liu, Jian Xu, Zhiyi Zhang, Guochun Wang, Xiaoxia Wang, Jiankang Hu, Hongbin Li, Zhuoli Zhang, Hongzhi Wang, Fuai Lu, Yingjue Du, Zenghui Xue, Yan Zhao, Zhanguo Li · 发表于：JAMA Network Open · 年份：2025 · DOI：10.1001/jamanetworkopen.2025.11095 · 被引用次数：14 · 研究领域：Rheumatoid Arthritis Research and Therapies、Biosimilars and Bioanalytical Methods、Spondyloarthritis Studies and Treatments

Importance: Treatment options are needed for patients with rheumatoid arthritis (RA) who experience inadequate response to conventional synthetic disease-modifying antirheumatic drugs (csDMARDs). The efficacy and safety profiles of subcutaneous tocilizumab monotherapy, tocilizumab combined with methotrexate, and methotrexate alone have not yet been evaluated in the same large-scale study. Objective: To evaluate the efficacy and safety of subcutaneous tocilizumab monotherapy, tocilizumab-methotrexate, and methotrexate alone for patients with RA. Design, Setting, and Participants: This double-blind, large-scale phase 3 randomized clinical trial was conducted at 19 sites in China from July 2017 through August 2022, with a 24-week double-blind treatment phase, a 24-week extension phase, and 8 additional weeks of safety follow-up. The trial included adults (aged 18-70 years) who had moderate to severe active RA and had experienced an inadequate response to csDMARDs. Interventions: In the 24-week double-blind treatment phase, patients were randomized 2:2:1 to tocilizumab-methotrexate, tocilizumab-placebo, or methotrexate-placebo. In the 24-week extension phase, patients who achieved a disease activity score using 28 joints (DAS28) of up to 3.2 at week 24 continued their double-blind phase treatment; those with scores greater than 3.2 received open-label tocilizumab-methotrexate. Tocilizumab (162 mg) was administered subcutaneously once every 2 weeks; oral methotrexate (10-25 mg) wa...