贝林妥欧单抗治疗儿童复发/难治急性淋巴细胞白血病的安全性及近期疗效分析

目的 分析贝林妥欧单抗治疗儿童复发/难治急性淋巴细胞白血病的安全性及近期疗效。方法 以2021年8月—2022年8月接受贝林妥欧单抗治疗的6例复发/难治急性淋巴细胞白血病患儿作为研究对象，回顾性分析患儿临床数据。结果 6例患儿中，男女各3例，中位入组年龄为10.5 （范围：5.0～13.0）岁。1例为难治性，经多次化疗均未缓解；5例为第一次复发，从诊断至复发中位间隔时间为30 （范围：9～60）个月。治疗前骨髓微小残留病为15.50 （范围：0.08～78.30）%。治疗后3例完全缓解，其中骨髓微小残留病转阴2例。5例患儿发生细胞因子释放综合征，其中1级3例，2级2例。4例桥接了异基因造血干细胞移植，贝林妥欧单抗治疗结束至移植中位间隔时间为50 （范围：40～70） d。6例患儿中位随访时间为170 d，总生存率为41.7%(95%CI:5.6%～76.7%)，中位生存时间为126 (95%CI:53～199) d。结论 贝林妥欧单抗治疗儿童复发/难治急性淋巴细胞白血病短期安全有效，长期疗效需扩大样本量进一步观察。中国当代儿科杂志，2023,25 (4):374-380]

Safety and short-term effectiveness of blinatumomab in the treatment of childhood relapsed/refractory acute lymphoblastic leukemia

Objective To study the safety and short-term effectiveness of blinatumomab in the treatment of childhood relapsed/refractory acute lymphoblastic leukemia (R/R-ALL).Methods Six children with R/R-ALL who received blinatumomab treatment from August 2021 to August 2022 were included as subjects, and a retrospective analysis was performed for their clinical data.Results Among the six children, there were three boys and three girls, with a median age of 10.5 (5.0-13.0) years at the time of inclusion. Of all six children, one had refractory ALL and did not achieve remission after several times of chemotherapy, and 5 relapsed for the first time, with a median time of 30 (9-60) months from diagnosis to relapse. Minimal residual disease (MRD) before treatment was 15.50% (0.08%-78.30%). Three children achieved complete remission after treatment, among whom two had negative conversion of MRD. Five children had cytokine release syndrome (CRS), among whom 3 had grade 1 CRS and 2 had grade 2 CRS. Four children were bridged to allogeneic hematopoietic stem cell transplantation, with a median interval of 50 (40-70) days from blinatumomab treatment to transplantation. The six children were followed up for a median time of 170 days, and the results showed an overall survival rate of 41.7% (95%CI: 5.6%-76.7%) and a median survival time of 126 (95%CI: 53-199) days.Conclusions Blinatumomab has good short-term safety and effectiveness in the treatment of childhood R/R-ALL, and its long-term effectiveness needs to be confirmed by studies with a larger sample size.