【摘要】 目的:探讨供者CD19嵌合抗原受体T细胞(chimeric antigen receptor T cell, CAR-T)治疗在儿童急性B淋巴细胞白血病(B-cell acute lymphoblastic leukemia, B-ALL)接受异基因造血干细胞移植后复发患者中的疗效及安全性。方法:选择2018年1月至2020年12月我中心收治的14例采用供者CD19 CAR-T细胞治疗的移植后复发B-ALL的临床资料，评估其疗效及安全性。结果:14例患者在回输后1个月内均获得骨髓微小残留病阴性的完全缓解。所有患者均发生细胞因子释放综合征，严重细胞因子释放综合征(≥3级)者9例(64.3%);4例发生免疫效应细胞相关神经毒性综合征，均经相应治疗后症状消失。中位随访时间为43.4(11.8～68.4)个月，5例复发，其中4例为移植后骨髓血液学复发患者，1例为移植后微小残留病水平复发患者，复发时CD19阳性2例，CD19阴性1例，另外2例表型不详，中位复发时间为13.4个月，3年总生存率为63%±13%,3年无白血病生存率为61%±14%。结论:供者CD19 CAR-T细胞治疗可有效治疗移植后复发B-ALL,但对回输前肿瘤负荷高的患者，建议再次缓解后桥接造血干细胞移植以改善长期生存情况。更多还原

【Abstract】 Objective: To investigate the efficacy and safety of donor CD19 chimeric antigen receptor T cell(CAR-T) therapy in pediatric patients with B-cell acute lymphoblastic leukemia(B-ALL) who relapsed after allogeneic hematopoietic stem cell transplantation. Methods: The clinical data of 14 patients with post-transplant relapsed B-ALL treated with donor CD19 CAR-T cell therapy were collected from January 2018 to December 2020, and the efficacy and safety of CAR-T cell therapy were evaluated. Results: All of the 14 patients obtained negative minimal residual disease within one month after infusion. Cytokine release syndrome occurred in all patients, with 9 patients(64.3%) developed severe cytokine release syndrome(grade ≥3), and 4 patients developed immune effector cell-associated neurotoxicity syndrome, all of which disappeared after corresponding treatment. The median follow-up time was 43.4(11.8-68.4) months, and there were 5 cases of recurrence with a median recurrence time of 13.4 months. Four cases were identified as bone marrow relapse prior to CAR-T cell therapy, and only one case was minimal residual disease recurrence. At relapse, the CD19 immunophenotype on leukemia blasts was CD19~+(2 cases), CD19~-(1 case), and CD19 unknown(2 cases). The 3-year overall survival rate was 63%±13%, and the 3-year leukemia-free survival rate was 61%±14%. Conclusion: Donor CD19 CAR-T cell therapy can effectively treat recurrent B-ALL after transplantation, but for patients with high tumor burden before infusion, it is recommended to bridge to hematopoietic stem cell transplantation after remission to improve long-term survival.更多还原