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

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

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。

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/RALL,and its long-term effectiveness needs to be confirmed by studies with a larger sample size.