为了探索抗CD19嵌合抗原受体基因修饰T（CD19-CAR-T）细胞治疗难治、复发的B急性淋巴白血病（B-acute lymphoblastic leukemia，B-ALL）安全有效的细胞剂量、毒副作用及其处理方法，2015年7月12日至2016年11月20日，笔者团队采用CD19-CAR-T细胞治疗了64例难治复发B-ALL患者。其中55例为原发耐药或难治性血液学复发患者，9例为FCM确认的难治性微小残留白血病(minimal residual disease,MRD )阳性B-ALL患者。在治疗工作早期， 2例死于治疗后相关并发症(treatment related motality, TRM)，疗效不可评估，4例未获得完全缓解(NR)。以后通过调整入组标准及回输细胞数，近期连续33例患者均获得完全缓解（CR），或达到CR但血液细胞计数未正常（CR incomplete, CRi)，无1例死亡。CD19-CAR-T细胞治疗达CR后，接受异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）者80%以上持续FCM-MRD阴性至观察结束，该疗效与第1次CR期(CR1)接受移植者相同；未移植者绝大多数在CR后1年内再次复发。治疗过程中，及时稳妥地处理好细胞因子释放综合征(cytokine releasing syndrome, CRS)及各种引起复发的因素是保证治疗成功的关键。

To explore the safety and effective doses of CD19 chimeric antigen receptor gene-modified T cells (CD19-CAR-T) for treating the refractory relapsed acute B lymphoblastic leukemia (R/R B-ALL)，as well as side effects and its optimal treatment methods, during July 12, 2015 to November 20, 2016 the 64 patients with R/R B-ALL were treated with the CD19-CAR-T. Among them 55 cases were recurrent primary drug-resistant or refractory patients with B-ALL, 9 cases were refractory positive minimal residual disease (MRD) patients with R/R-ALL confirmed by flow cytometry (FCM). In the early period, the 2 patients dies from treatment-related complications and their efficacy could not evaluated, and the 4 patients did not achieve complete remission (CR). After adjusting inclusion criteria and retransfusion cell numbers of CD19-CAR-T, recently the 33 patients continuously achieved RC, or achieved RC but numbers of their blood cells were not normal without any dead cases. More than 80% patients, who received allogeneic hematopoietic stem cell transplantation (allo-HSCT) after treatment with CD19-CAR-T, continued negative MRD confirmed by FCM until end of the observation, of which efficacy was the same as that of the patients received allo-HSCT at first CR time, and most of the patients who did not receive allo-HSCT had a relapse again within 1 year after CR. In course of the treatment, keys to ensure success of the treatment should be timely and safely handling of cytokines releasing syndromes and various factors of inducing recurrent.