T细胞急性淋巴细胞白血病（T-ALL）在治疗后复发或耐药时很难治疗；复发或难治性T-ALL患者的预后普遍较差。我们报告了一个由 17 名此类患者组成的病例系列，他们接受了表达抗 CD7 CAR 的自体嵌合抗原受体 (CAR) T 细胞和抗 CD7 蛋白表达阻断剂 (PEBL)，从而防止了 CAR T 细胞自相残杀。尽管白血病负荷高且 CAR T 细胞剂量低，17 名患者中的 16 名在 1 个月内达到了微小残留病阴性完全缓解。另一名患者在输注前有 CD7-T-ALL 细胞，输注后仍持续存在。毒性较轻：10 名患者出现 1 级细胞因子释放综合征，3 名患者出现 2 级细胞因子释放综合征；两名患者出现 1 级免疫效应细胞相关神经毒性综合征。 11 名患者保持无复发（中位随访 15 个月），其中包括所有接受同种异体移植的 9 名患者。第一个患者在输注后 55 个月达到缓解，无需进一步化疗或移植；循环 CAR T 细胞可检测到 2 年。淋巴细胞清除后再生的 T 细胞缺乏 CD7 表达，是多克隆的，并对 SARS-CoV-2 疫苗接种有反应；随着 CAR T 细胞的消失，CD7 免疫细胞重新出现。总之，自体抗 CD7 PEBL-CAR T 细胞具有强大的抗白血病活性，可能是治疗 T-ALL 的有效选择。© 2024。作者获得 Springer Nature America, Inc. 的独家许可。

T cell acute lymphoblastic leukemia (T-ALL) is difficult to treat when it relapses after therapy or is chemoresistant; the prognosis of patients with relapsed or refractory T-ALL is generally poor. We report a case series of 17 such patients who received autologous chimeric antigen receptor (CAR) T cells expressing an anti-CD7 CAR and an anti-CD7 protein expression blocker (PEBL), which prevented CAR T cell fratricide. Despite high leukemic burden and low CAR T cell dosing, 16 of the 17 patients attained minimal residual disease-negative complete remission within 1 month. The remaining patient had CD7- T-ALL cells before infusion, which persisted after infusion. Toxicities were mild: cytokine release syndrome grade 1 in ten patients and grade 2 in three patients; immune effector cell-associated neurotoxicity syndrome grade 1 in two patients. Eleven patients remained relapse-free (median follow-up, 15 months), including all nine patients who received an allotransplant. The first patient is in remission 55 months after infusion without further chemotherapy or transplantation; circulating CAR T cells were detectable for 2 years. T cells regenerating after lymphodepletion lacked CD7 expression, were polyclonal and responded to SARS-CoV-2 vaccination; CD7+ immune cells reemerged concomitantly with CAR T cell disappearance. In conclusion, autologous anti-CD7 PEBL-CAR T cells have powerful antileukemic activity and are potentially an effective option for the treatment of T-ALL.© 2024. The Author(s), under exclusive licence to Springer Nature America, Inc.