Blinatumomab has remarkable efficacy in patients with relapsed/refractory (r/r) or measurable residual disease (MRD)-positive B-cell acute lymphoblastic leukemia (B-ALL). In many patients, blinatumomab treatment is followed by allogeneic hematopoietic stem cell transplantation (HSCT). However, the influence of blinatumomab on HSCT outcomes in children and young adults (YA) remains to be fully elucidated. We conducted a single-center, retrospective analysis on patients given blinatumomab as last treatment before HSCT. Seventy-eight pediatric and YA patients were evaluated. With a median follow-up of 23.23 months, the 2-year disease-free (DFS) and overall survival (OS) probability were 72.2% and 89.2%, respectively, with a 2-year cumulative incidence (CI) of non-relapse mortality (NRM) of 2.6%. A trend toward improved 2-year DFS, but not OS, was noted in patients transplanted in first complete remission (CR1) (92.9%) compared to those in second or greater remission (CR2/3) (68.5%, p=0.18) due to a lower CI of relapse (0% vs. 29.9%, p=0.05). Among CR2/3 patients, those receiving the sequential combination of inotuzumab and blinatumomab had a significantly lower CI of relapse as compared to those who did not receive inotuzumab (9.5% vs. 40.4%, p=0.023). Relapse after HSCT occurred in 16 patients, all exhibiting CD19-positive blasts; 10 of them received anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy and 2 inotuzumab as salvage therapy, leading to a 2-year post-relapse OS of 52.7%. Our results indicate that HSCT following blinatumomab in children and YA with B-ALL is highly effective, being associated with low NRM and not affecting the efficacy of subsequent salvage immunotherapies, including CAR-T cells.

中文翻译：

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患有 B 细胞急性淋巴细胞白血病的儿童和年轻人在同种异体造血干细胞移植前接受 blinatumomab 作为最后一次巩固治疗的结果。


Blinatumomab 对复发/难治性 （r/r） 或可测量残留病 （MRD） 阳性 B 细胞急性淋巴细胞白血病 （B-ALL） 患者具有显着疗效。在许多患者中，blinatumomab 治疗后进行同种异体造血干细胞移植 （HSCT）。然而，blinatumomab 对儿童和年轻人 （YA） HSCT 结果的影响仍有待完全阐明。我们对 HSCT 前最后一次治疗 blinatumomab 的患者进行了单中心回顾性分析。对 78 例儿科和 YA 患者进行了评估。中位随访 23.23 个月，2 年无病 （DFS） 和总生存期 （OS） 概率分别为 72.2% 和 89.2%，非复发死亡率 （NRM） 的 2 年累积发生率 （CI） 为 2.6%。与第二次或更高缓解期 （CR2/3） 患者 （68.5%，p=0.18） 相比，第一次完全缓解 （CR1） 移植的患者 （92.9%） 有改善 2 年 DFS 但没有 OS 的趋势，因为复发 CI 较低 （0% vs. 29.9%，p=0.05）。在 CR2/3 患者中，与未接受 inotuzumab 的患者相比，接受 inotuzumab 和 blinatumomab 序贯联合治疗的患者的复发 CI 显著降低 （9.5% vs. 40.4%，p=0.023）。16 例患者 HSCT 后复发，均表现出 CD19 阳性原始细胞;其中 10 例接受抗 CD19 嵌合抗原受体 T 细胞 （CAR-T） 治疗和 2 例 inotuzumab 作为挽救治疗，导致复发后 2 年 OS 为 52.7%。我们的结果表明，儿童和 YA 伴 B-ALL 的 blinatumomab 后 HSCT 非常有效，与低 NRM 相关，并且不会影响后续挽救免疫疗法（包括 CAR-T 细胞）的疗效。