Objective: To study the safety and effectiveness of humanized CD19-targeted CAR-T cells (hCART19s) for treatment of patients with refractory/relapsed (R/R) B-ALL.
Methods: The analyzed patients were 15 children and adults with relapsed/refractory B-ALL who not received treatment with murine CD19 CAR-T cells. The patients received a single dose (1×106/kg) of autologous hCART19 infusion after lymphodepletion chemotherapy based on cyclophosphamide and fludarabine.
Results: Among the 15 patients, 13/14 (92.9%) evaluable patients achieved complete remission (CR) or CR with incomplete recovery of blood cells (CRi) on day 30 after hCART19s infusion. At day 180 after the infusion, the overall survival rate was 73.3%, and the leukemia-free survival rate was 69.2%. The cumulative incidence of relapse was 24.5% and non-relapse mortality rate was 7.7%. During treatment,12/15 patients (80%) developed cytokine release syndrome (CRS) of grade 1-2, and 3 patients (20.0%) developed CRS of grade 3-5. Only one patient (6.7%) suffered from the reversible neurotoxicity.
Conclusion: hCART19s can effectively treat refractory/relapsed (R/R) adult and children with B-ALL, and the incidence of treatment-related CRS and neurotoxicity is low.
题目: 人源化抗CD19嵌合抗原受体T细胞治疗急性B淋巴细胞白血病的临床研究.
目的: 研究人源化CD19 CAR-T细胞治疗难治/复发性B-ALL患者的安全性和有效性.
方法: 入组的患者为15例既往未接受鼠源CD19 CAR-T细胞治疗的复发/难治的 B-ALL儿童和成人患者。患者在氟达拉滨和环磷酰胺为基础的预处理化疗后，接受了1×106/kg的自体人源化CD19 CAR-T（hCART19）细胞的输注。在患者回输CAR-T细胞后观察其治疗的安全性及有效性.
结果: 15名患者中，13/14（92.9％）可评价的患者在hCART19输注后30 d获得了完全缓解（CR）或者CR伴血细胞不完全恢复（CRi）。180 d时总生存率及无病生存率分别达到73.3%和69.2%。累积复发率及非复发死亡率分别是24.5% 和7.7%。15例患者中有12名患者（80%）发生了1-2级的细胞因子释放综合征（CRS），3名患者（20.0%）发生了3-5级的CRS，而只有1名患者（6.7%）发生了可逆性神经毒性.
结论: hCART19s可有效治疗复发/难治的成人及儿童B-ALL患者，且治疗相关的CRS及神经毒性发生率较低.