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

李晓兰, 刘立鹏, 刘芳, 郭晔, 陈晓娟, 竺晓凡, 杨文钰

中国当代儿科杂志 ›› 2023, Vol. 25 ›› Issue (4) : 374-380.

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中国当代儿科杂志 ›› 2023, Vol. 25 ›› Issue (4) : 374-380. DOI: 10.7499/j.issn.1008-8830.2210114
论著·临床研究

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

  • 李晓兰, 刘立鹏, 刘芳, 郭晔, 陈晓娟, 竺晓凡, 杨文钰
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Safety and short-term effectiveness of blinatumomab in the treatment of childhood relapsed/refractory acute lymphoblastic leukemia

  • LI Xiao-Lan, LIU Li-Peng, LIU Fang, GUO Ye, CHEN Xiao-Juan, ZHU Xiao-Fan, YANG Wen-Yu
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摘要

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]

Abstract

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. Citation:Chinese Journal of Contemporary Pediatrics, 2023, 25(4): 374-380

关键词

复发/难治急性淋巴细胞白血病 / 贝林妥欧单抗 / 安全性 / 近期疗效 / 儿童

Key words

Relapsed/refractory acute lymphoblastic leukemia / Blinatumomab / Safety / Short-term effectiveness / Child

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导出引用
李晓兰, 刘立鹏, 刘芳, 郭晔, 陈晓娟, 竺晓凡, 杨文钰. 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[J]. 中国当代儿科杂志. 2023, 25(4): 374-380 https://doi.org/10.7499/j.issn.1008-8830.2210114
LI Xiao-Lan, LIU Li-Peng, LIU Fang, GUO Ye, CHEN Xiao-Juan, ZHU Xiao-Fan, YANG Wen-Yu. Safety and short-term effectiveness of blinatumomab in the treatment of childhood relapsed/refractory acute lymphoblastic leukemia[J]. Chinese Journal of Contemporary Pediatrics. 2023, 25(4): 374-380 https://doi.org/10.7499/j.issn.1008-8830.2210114

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