Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial

作者：Yunfei Chen, Yanmei Xu, Jiawen Dai, Ting Sun, Huiyuan Li, Zhengjie Hua, Zeping Zhou, Hu Zhou, Zhenyu Yan, Xingli Zhao, Feng Xue, Wei Liu, Xiaofan Liu, Rongfeng Fu, Wentian Wang, Ying Chi, H. Dong, Mankai Ju, Xinyue Dai, Wenjing Gu, Xiaolei Pei, Renchi Yang, Lei Zhang · 发表于：BMJ · 年份：2025 · DOI：10.1136/bmj-2025-084314 · 被引用次数：6 · 研究领域：Platelet Disorders and Treatments、Heparin-Induced Thrombocytopenia and Thrombosis、Blood disorders and treatments

Abstract Objective To assess the efficacy and safety of CM313, an anti-CD38 monoclonal antibody, in adults with persistent or chronic primary immune thrombocytopenia. Design Multicentre, randomised, placebo controlled, phase 2 trial. Setting Five hospitals in China, 16 January to 11 June 2024. Participants 45 patients aged ≥18 years with persistent or chronic immune thrombocytopenia who failed to respond to, or relapsed after, glucocorticoid treatment but had previously responded to standard first line treatment. Interventions Patients were randomised to receive intravenous CM313 (16 mg/kg) or placebo weekly for eight weeks. Main outcome measures The primary outcome was overall response rate (at least two consecutive platelet counts ≥30×10 9 /L, a minimum doubling from baseline, and no bleeding) at week 8. A secondary outcome was time to the first two consecutive platelet counts ≥50×10 9 /L. Results Of 56 patients screened, 45 were randomised to receive CM313 (n=30) or placebo (n=15). At week 8, the overall response rate was higher in the CM313 group (83%; 25/30) compared with placebo group (20%; 3/15): difference 63.3% (95% confidence interval 33.7% to 81.3%; P<0.001). The median time to a platelet count of ≥50×10 9 /L was one week in the CM313 group but was not reached in the placebo group (P<0.001). The median cumulative response duration for platelet counts ≥50×10 9 /L was 18 weeks in the CM313 group versus three weeks in the placebo group (P=0.004). Treatment emerg...