Daratumumab in patients with immune thrombocytopenia: a single-center, open-label, phase 2 trial

作者：Yunfei Chen, Zhengjie Hua, Yanmei Xu, Jia Chen, Qing Wen, Ting Sun, Huiyuan Li, Xiaofan Liu, Rongfeng Fu, Mankai Ju, Feng Xue, Wei Liu, Huan Dong, Wenjing Gu, Xinyue Dai, Wentian Wang, Ying Chi, Xiaolei Pei, Renchi Yang, Lei Zhang · 发表于：EClinicalMedicine · 年份：2026 · DOI：10.1016/j.eclinm.2026.103849 · 被引用次数：1 · 研究领域：Platelet Disorders and Treatments、Heparin-Induced Thrombocytopenia and Thrombosis、Blood groups and transfusion

Background Immune thrombocytopenia is an autoimmune bleeding disorder. Daratumumab, a human monoclonal antibody targeting CD38 glycoprotein, is currently under clinical investigation as a potential therapy for autoimmune diseases. This trial aimed to evaluate the efficacy and safety of daratumumab in adults with immune thrombocytopenia. Methods This single-center, open-label, phase 2 trial enrolled patients with a platelet count <30 × 10 9 platelets per L during the screening period. The study protocol comprised a 4-week screening phase, an 8-week treatment course, and a 16-week follow-up period. Daratumumab was administered intravenously at 16 mg/kg once weekly for eight consecutive weeks. The primary endpoint was defined as two or more consecutive platelet counts of at least 50 × 10 9 platelets per L within 8 weeks. This trial is registered at clinicaltrials.gov as #NCT05562882. Findings Between December 10, 2022, and August 15, 2024, 63 patients were enrolled. All participants completed the full treatment and follow-up schedule. 52 patients (83%) reached the primary endpoint, with 46 (73%) having platelet counts of at least 50 × 10 9 platelets per L within the first week. At week 8, 49 patients (78%) had an overall response, including a complete response in 32 (51%). An overall response was observed in 38 patients (60%) at week 24. The proportion of patients with bleeding symptoms (WHO grade 1–4) declined from 44% (28 of 63) at baseline to 10% (6 of 63) at week 8. Infusion...