难治复发急性淋巴细胞白血病（acute lymphocytic leukemia， ALL）预后差，生存期短，其治疗已成为国际难题。嵌合抗原受体基因修饰T（chimeric antigen receptor gene-modified T，CAR-T）细胞是目前最具应用前景的靶向免疫治疗，靶向CD19的CAR-T细胞（CD19-CAR-T）治疗儿童及成人难治复发性ALL的完全缓解率（complete remission,CR）可达90%以上，疗效远高于化疗。然而，细胞因子释放综合征（cytokine release syndrome，CRS）、严重神经毒性（serious neurotoxicity，SNT）、脱靶效应以及疾病复发等严重限制了CAR-T细胞的进一步临床应用。本文主要阐述CAR-T细胞的制备技术、预处理方案、细胞输注剂量及各种并发症防治策略等最新研究进展。

Patients with refractory/relapsed acute lymphoblastic leukemia (ALL) have poor prognosis and short survival time, which has become an international conundrum. Chimeric antigen receptor gene-modified T cell (CAR-T) therapy has been the most promising application targeted immunotherapy so far. CD19-targeted CAR-T (CD19-CAR-T) therapy could reach more than 90% of complete remission rate (CR) for the children and adults with refractory/relapsed ALL, of which efficacy was much higher than that of chemotherapy. However, complications, such as cytokine release syndrome (CRS), serious neurotoxicity (SNT), off-target effect and relapse, seriously impeded its further clinical application. This article mainly reviewed the latest progresses of researches on the manufacture technologies, the conditioning regimens, the cell infusion doses and the prevention and treatment strategies of complications for the CAR-T therapy.

国家重点基础研究发展计划(973计划)基金项目（No.2015CB964900）