Long-term efficacy and safety of low-dose rituximab in immune thrombocytopenia: a multicentre, prospective, open-label, randomised controlled trial

作者：Yunfei Chen, Jiaying Liu, Jiawen Dai, Ting Sun, Zijian Qiao, Maosheng Wang, Hu Zhou, Zeping Zhou, Xiaofan Liu, Rongfeng Fu, Feng Xue, Wei Liu, Mankai Ju, Huan Dong, Xinyue Dai, Wenjing Gu, Renchi Yang, Lei Zhang · 发表于：Annals of Hematology · 年份：2026 · DOI：10.1007/s00277-026-06908-2 · 被引用次数：1 · 研究领域：Platelet Disorders and Treatments、Heparin-Induced Thrombocytopenia and Thrombosis、Blood groups and transfusion

Around 60–80% of patients with immune thrombocytopenia (ITP) experience disease relapse after stopping corticosteroids. Rituximab (RTX) is an effective second-line treatment. Low-dose RTX has shown similar efficacy to standard dose, but the optimal dosage remains uncertain. This multicentre, prospective, open-label, randomised controlled trial aimed to compare the long-term efficacy (5-year follow-up) and 1-year safety of two low-dose RTX regimens in Chinese adult patients with glucocorticoid-resistant/dependent or relapsed ITP. Patients (n = 104) were randomised 1:1 to group A (RTX 100 mg weekly for 4 weeks) or group B (RTX 375 mg/m2 once). The primary outcome was overall response (OR) at 3 months after RTX initiation. Response was followed up until disease relapse or for 5 years. Forty-nine patients in Group A and 51 patients in Group B completed the intervention. At 3 months after RTX initiation, 32 patients in group A (65.3%) and 33 patients in group B (64.7%) achieved OR, and the complete response rate was 42.9% (21/49) in group A and 39.2% (20/51) in group B. The sustained response rates at 6 months, 1, 2, and 5 years were 59.2%, 42.9%, 28.6%, and 20.4% in group A and 58.8%, 43.1%, 33.3%, and 17.6% in group B. These variables were not statistically different between two groups. The most common adverse events were upper respiratory tract and pulmonary infections. RTX 375mg/m2 once showed similar long-term efficacy compared to RTX 100 mg weekly for 4 weeks, with a compara...