Updated data of CLL1 CAR-T cell therapy in adult patients with relapsed/refractory acute myeloid leukemia

作者：Xiaomei Zhang, Wenyi Lu, Wenjun Zhang, Xiaoyuan He, Yu Zhang, Xin Jin, Meng Zhang, Yingshuai Wang, Xuwen Guan, Rui Zhang, Bing Wang, Hairong Lyu, Xia Xiao, Xue Bai, Yedi Pu, Juanxia Meng, Haibo Zhu, Zhao Wang, Huan Zhang, Huan Zhang, Cuicui Lyu, Hongkai Zhang, Hongkai Zhang, Aibin Liang, Mingfeng Zhao · 发表于：Journal of Hematology & Oncology · 年份：2025 · DOI：10.1186/s13045-025-01764-5 · 被引用次数：7 · 研究领域：CAR-T cell therapy research、Acute Lymphoblastic Leukemia research、Chronic Lymphocytic Leukemia Research

CLL1-targeted chimeric antigen receptor T (CAR-T) cell therapy has shown clinically meaningful activity in relapsed/refractory acute myeloid leukemia (R/R AML). This updated phase I study enrolled 38 adults with R/R AML to evaluate the safety and efficacy of this treatment according to the prespecified protocol. Treatment-related adverse events included grade 3/4 cytokine release syndrome (CRS) in 17 patients (44.74%) and grade 4 immune effector cell-associated neurotoxicity syndrome (ICANS) in 1 patient (2.63%). Persistent cytopenia (all grades) was observed in all patients, with a median time to neutrophil recovery of 45 days. At a median follow-up of 24.87 months, the objective response rate (ORR) was 73.68% (28/38), and the minimal residual disease-negative complete response rate (MRD−CR) was 42.11%. Median progression-free survival (PFS) and overall survival (OS) were 9 months (95%CI 3.03–14.47) and 12.17 months (95%CI 3.03–24.87), respectively. The two-year PFS and OS rates were 47.94% (95%CI: 32.00%-63.83%) and 51.43% (95%CI 35.51%-67.32%), respectively. These results indicate a clinically relevant antitumor efficacy and a manageable safety profile of CLL1-targeted CAR-T cell therapy in adults with R/R AML. Registry: www.chictr.org.cn , TRN: ChiCTR2000041054, Registration date: 17 December 2020.