Autologous CD19-directed CAR-T exhibit promising safety and efficacy in refractory primary immune thrombocytopenia

作者：Mengli Xu, Shiqi Li, Zhi Yang, Lin Liu, Zhongtao Yuan, Yu Li, Yingzi Zhang, Hong Juan, Lihui Peng, Xuejuan Zhang, Lihua Zhang, Yingnian Chen, Qingying Zang, Qianzhen Zhang, Junjie Shen, Xiaoping Li, Yunyan Li, Yancheng Dong, Youcheng Wang, Dingsong Zhang, Linling Wang, Meiling Wang, Wei Zhu, Jiong Hu, Cheng Qian, Sanbin Wang · 发表于：Blood · 年份：2025 · DOI：10.1182/blood-2025-4175 · 被引用次数：3 · 研究领域：CAR-T cell therapy research、Monoclonal and Polyclonal Antibodies Research、Platelet Disorders and Treatments

Abstract Background: Refractory primary immune thrombocytopenia (ITP) patients are characterized by great treatment difficulties, markedly reduced quality of life, and high hemorrhagic or infectious morbidity and mortality. New promising strategies are needed. Anti-CD19 Chimeric antigen receptor (CAR) T cell therapy, which can deeply eliminate CD19 positive B cells in circulation and tissues, has demonstrated revolutionary efficacy in autoimmune diseases, especially in SLE-associated ITP and multi-refractory primary ITP. We reported the preliminary safety and efficacy of autologous CD19-targeted CAR-T in the treatment of three refractory primary ITP patients. Here, we present updated data. Methods: From February 2024 to June 2025, four eligible refractory primary ITP patients were enrolled. The eligibility details are listed in the clinicaltrials.gov as register number NCT06352281. The patients received conventional three-day FC lymphodepletion preconditioning chemotherapy with fludarabine 30mg/m2 and cyclophosphamide 300mg/m2 on days -5, -4, and -3 except one patient. Then, a single dose of 1.5×105/kg or 3×105/kg CAR-T cells were infused intravenously. As to safety profile, cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were graded by ASTCT criteria, and other adverse events were evaluated according to Common Terminology Criteria for Adverse Events (CTCAE) 5.0. Clinical efficacy was evaluated based on the platelet counts an...