IGF-1R Inhibitor IBI311 for the Treatment of Active Thyroid Eye Disease in Chinese Patients

作者：Haiyang Zhang, Jing Sun, Yinwei Li, Limin Zhu, Zhongyan Shan, Wei Lu, Jie Shen, Yuhong Shi, Liming Tao, Hongwei Jiang, Linong Ji, Yuman Zhu, Hao Gu, Xiaowei Liu, Bin Sun, Yanjie Tian, Qinghuai Liu, Jianbo Zhou, Hong Zhao, Haixia Guan, Lei Zhu, Junping Wen, Yingli Lu, Jie Qiao, Wen Zhang, Junjie Deng, Shujie Lu, Han Han‐Zhang, Lei Qian, Huifang Zhou, Xianqun Fan · 发表于：JAMA Ophthalmology · 年份：2025 · DOI：10.1001/jamaophthalmol.2025.3350 · 被引用次数：10 · 研究领域：Ophthalmology and Eye Disorders、Glaucoma and retinal disorders、Thyroid Disorders and Treatments

Importance: Thyroid eye disease (TED), a disfiguring and potentially sight-threatening condition with racial phenotypic variations, currently has limited effective treatments. Insulin-like growth factor 1 receptor (IGF-1R) inhibitors therapy has emerged as a promising treatment option, although it remains less accessible and lacks substantial evidence in Asian patients. Objective: To assess efficacy and safety of IBI311, an IGF-1R inhibitor with an identical amino acid sequence to teprotumumab but a different dosage form, in Chinese patients with active TED. Design, Setting, and Participants: This was a randomized, double-masked, placebo-controlled, multicenter, 24-week phase 3 trial with recruitment conducted across 20 tertiary hospitals in China from May to December 2023. Chinese participants with active (clinical activity score [CAS] ≥3) moderate to severe TED were included after excluding individuals with active TED onset over 270 days; sight-threatening TED; or history of steroid pulse therapy, radiotherapy, or surgery for TED. Interventions: Eighty-two participants were randomized 2:1 to receive intravenous infusions of either IBI311 or placebo once every 3 weeks for 21 weeks with follow-up through week 24. Main Outcomes and Measures: The primary outcome was the proptosis response rate (proptosis reduction ≥2 mm) in the study eye at week 24. Results: Participants (mean [SD] age, 39.6 [10.9] years; 56 [68.3%] women) were randomized to receive IBI311 (n = 54) or placebo (...