Objective: This study aimed to examine the safety and efficacy of CD19 chimeric antigen receptor T cell (CD19 CAR-T) therapy in relapsed/refractory Philadelphia chromosome-positive acute B-precursor lymphoblastic leukemia (R/R Ph(+) B-ALL) . Methods: The clinical data of 14 patients with R/R Ph(+) B-ALL treated with CD19 CAR-T cell therapy from November 2016 to April 2019 were retrospectively analyzed. Results: Among the 14 patients in this study, 7 were male and 7 were female, with a median age of 33 (7-66) years old. The efficacy was evaluated on the 28th day following CAR-T cells infusion; the overall response rate was 100.0% (14/14) , the complete response (CR) rate was 92.9% (13/14) , and the partial response (PR) rate was 7.1% (1/14) . After CAR-T cells infusion,12 cases (85.7%) developed cytokine release syndrome (CRS) : 1 case of grade 1 CRS, 4 cases of grade 2 CRS, 6 cases of grade 3 CRS, and 1 case of grade 4 CRS. Moreover, one case developed CAR T-cell-related encephalopathy syndrome (CRES) ; 14 cases had Ⅲ-Ⅳ hematological toxicity; and 13 CR cases had B cell dysplasia. These adverse reactions were all controllable. The median follow-up time was 441 (182-923) d. The median overall survival (OS) and progression-free survival (PFS) were 515 [95% confidence interval (CI) 287-743] days and 207 (95% CI 123-301) days, respectively. Conclusion: CD19 CAR-T cell therapy is safe and effective for R/R Ph(+) B-ALL treatment. However, the long-term efficacy needs to be further improved.
目的： 研究靶向CD19的嵌合抗原受体T细胞（CAR-T细胞）治疗复发难治Ph染色体阳性急性B淋巴细胞白血病（R/R Ph(+) B-ALL）的安全性及有效性。 方法： 对2016年11月至2019年4月采用CD19 CAR-T细胞治疗的14例R/R Ph(+) B-ALL患者的临床资料进行回顾性分析。 结果： 14例患者中，男7例，女7例，中位年龄33（7~66）岁。输注CAR-T细胞后第28天近期疗效：总反应率（ORR）为100.0%（14/14），其中完全缓解（CR）率为92.9%（13/14）；部分缓解（PR）率为7.1%（1/14）。CAR-T细胞治疗后有12例（85.7%）发生细胞因子释放综合征（CRS），其中1级1例、2级4例、3级6例、4级1例；1例（例3）发生CAR-T细胞相关脑病综合征（CRES）；全部14例患者均有Ⅲ~Ⅳ级血液学不良反应；13例CR患者均发生B细胞发育不全。上述不良反应均可控。中位随访441（182~923）d，中位总生存期、无进展生存期分别515（95% CI 287~743）d、207（95% CI 123~301）d。 结论： CD19 CAR-T细胞治疗复发难治性Ph(+) B-ALL安全有效，短期疗效显著，长期疗效有待进一步提高。.
Keywords: Chimeric antigen receptor T cell, CD19; Leukemia, B-precursor lymphoblastic, acute; Philadelphia chromosome; Tyrosine kinase inhibitors.