Xie Jiao, Liu Suxiang, Zhou Ming, Wang Yi, He Hailong, Xiao Peifang, Hu Shaoyan, Lu Jun
Department of Hematology and Oncology, Children's Hospital of Soochow University, Suzhou, China.
Front Pediatr. 2023 Aug 4;11:1187607. doi: 10.3389/fped.2023.1187607. eCollection 2023.
To evaluate the clinical efficacy and safety of a short course of blinatumomab in children with refractory or relapsed precursor B-cell acute lymphoblastic leukemia (R/R-BCP-ALL).
The clinical data of 33 R/R BCP-ALL children aged 0-18 years who underwent a short course of blinatumomab (14 days) between August 2021 and November 2022 were retrospectively collected and analyzed.
Among 33 patients with BCP-ALL, 26 achieved complete remission (CR), with a total remission rate of 78.8% (26/33). The duration of remission was approximately 14 days. Of the 7 children without CR, 5 were still in remission at 28 days. In 11 patients with refractory disease and 22 with recurrence, the remission rates were 90.9% (10/11) and 72.7% (16/22), respectively. The overall survival (OS) rates of the 26 patients with CR and seven patients without CR were 96.1% and 57.1% ( = 0.002), respectively, and the disease-free survival (DFS) rates were 96.1% and 42.9% ( < 0.001), respectively. Among the 26 patients with CR, 15 underwent bridging hematopoietic stem cell transplantation (HSCT) and 11 did not receive HSCT; with OS rates of 93.3% and 100% ( = 0.40) and DFS rates of 93.3% and 100% ( = 0.400), respectively. The OS for all patients was 87.9% (29/33) and the DFS was 84.8% (28/33). There were 18 cases (54.5%) of cytokine release syndrome (CRS), 2 cases (6.1%) of severe CRS (all grade 3), 1 case (3.0%) of immune effector cell-associated neurotoxicity syndrome (ICANS), 0 cases (0%) of ICANS ≥ grade 3, and no deaths caused by treatment.
Short-term follow-up revealed a high R/R BCP-ALL remission rate in children treated with a short course of blinatumomab. The toxicity was low and controllable. No significant short-term survival benefits were observed after bridging HSCT with blinatumomab. In developing countries, a short course of blinatumomab can achieve satisfactory outcomes, while reducing household costs and saving medical resources.
评估短期使用博纳吐单抗治疗难治性或复发性前体B细胞急性淋巴细胞白血病（R/R-BCP-ALL）患儿的临床疗效和安全性。
回顾性收集并分析2021年8月至2022年11月期间33例年龄在0至18岁接受短期（14天）博纳吐单抗治疗的R/R BCP-ALL患儿的临床资料。
33例BCP-ALL患者中，26例达到完全缓解（CR），总缓解率为78.8%（26/33）。缓解持续时间约为14天。7例未达到CR的患儿中，5例在28天时仍处于缓解状态。11例难治性疾病患者和22例复发性患者的缓解率分别为90.9%（10/11）和72.7%（16/22）。26例CR患者和7例未CR患者的总生存（OS）率分别为96.1%和57.1%（P=0.002），无病生存（DFS）率分别为96.1%和42.9%（P<0.001）。26例CR患者中，15例接受了桥接造血干细胞移植（HSCT），11例未接受HSCT；OS率分别为93.3%和100%（P=0.40），DFS率分别为93.3%和100%（P=0.400）。所有患者的OS为87.9%（29/33），DFS为84.8%（28/33）。有18例（54.5%）发生细胞因子释放综合征（CRS），2例（6.1%）为严重CRS（均为3级），1例（3.0%）发生免疫效应细胞相关神经毒性综合征（ICANS），0例（0%）ICANS≥3级，且无治疗相关死亡。
短期随访显示，短期使用博纳吐单抗治疗的R/R BCP-ALL患儿缓解率高。毒性低且可控。博纳吐单抗桥接HSCT后未观察到显著的短期生存获益。在发展中国家，短期使用博纳吐单抗可取得满意疗效，同时降低家庭成本并节省医疗资源。
