基于 MRD 指导治疗，比较 CAR-T 细胞治疗后或化疗后单倍体造血干细胞移植在首次复发 B 细胞急性淋巴细胞白血病患儿中的长期生存与安全性

Measurable residual disease (MRD) positivity before haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is an independent prognostic factor in determining outcomes in patients with B-cell acute lymphoblastic leukemia (ALL). In this study, we conducted a parallel comparison of the efficacy and safety in patients with suboptimal MRD response after reinduction who underwent haplo-HSCT after chimeric antigen receptor T-cell (CAR-T) therapy or chemotherapy. Forty B-cell ALL patients who relapsed after first-line chemotherapy and with an MRD 0.

1% after reinduction were analyzed. The median pre-HSCT MRD in the CAR-T group ( n = 26) was significantly lower than that in the chemotherapy group ( n = 14) (0. 009% vs. 0. 3%, p = 0. 006). The CAR-T group exhibited a trend toward improved 3-year leukemia-free survival and a significantly improved 3-year overall survival compared to the chemotherapy group [71. 8% (95% confidence interval (CI): 53. 9-89. 6) vs. 44. 4% (95% CI: 15. 4-73. 4), p = 0. 19 and 84. 6% (95% CI: 70. 6-98. 5) vs. 40. 0% (95% CI: 12. 7-67. 2), p = 0. 008; respectively].

Furthermore, no increased risk of graft-versus-host disease, treatment-related mortality, or infection was observed in the CAR-T group.

Our study suggests that CAR-T therapy effectively eliminates pre-HSCT MRD, resulting in better survival in the context of haplo-HSCT.