研究动态
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儿童复发/难治性B前体急性淋巴细胞白血病的短程布雷纳单抗治疗.

Short-course blinatumomab for refractory/relapse precursor B acute lymphoblastic leukemia in children.

发表日期:2023
作者: Jiao Xie, Suxiang Liu, Ming Zhou, Yi Wang, Hailong He, Peifang Xiao, Shaoyan Hu, Jun Lu
来源: Stem Cell Research & Therapy

摘要:

评估在难治性或复发性B前体细胞急性淋巴细胞白血病(R/R-BCP-ALL)儿童中,短程布利纳单抗的临床疗效和安全性。于2021年8月至2022年11月间,回顾性收集和分析了33名接受短程布利纳单抗治疗(14天)的R/R BCP-ALL患儿(年龄0-18岁)的临床数据。在33名BCP-ALL患者中,26名患者达到完全缓解(CR),总缓解率为78.8%(26/33)。缓解持续时间约为14天。7名未达到CR的儿童中,5名在28天仍处于缓解状态。11名难治性疾病患者和22名复发性疾病患者中,缓解率分别为90.9%(10/11)和72.7%(16/22)。26名CR患者和7名未达到CR的患者的总生存率(OS)分别为96.1%和57.1%(p = 0.002),无病生存率(DFS)分别为96.1%和42.9%(p < 0.001)。在26名CR患者中,15名接受了造血干细胞移植(HSCT)和11名未接受HSCT;其OS分别为93.3%和100%(p = 0.40),DFS分别为93.3%和100%(p = 0.400)。所有患者的总体生存率(OS)为87.9%(29/33),DFS为84.8%(28/33)。共有18例(54.5%)细胞因子释放综合征(CRS),2例(6.1%)严重CRS(均为3级),1例(3.0%)免疫效应细胞相关的神经毒性综合征(ICANS),0例(0%)ICANS ≥ 3级,无因治疗导致的死亡。短期随访显示,在接受短程布利纳单抗治疗的儿童中,R/R BCP-ALL的缓解率较高。毒性低且可控。在布利纳单抗桥接HSCT后没有观察到明显的短期生存优势。在发展中国家,短程布利纳单抗可以取得满意的疗效,同时减少家庭开支并节约医疗资源。© 2023 Xie, Liu, Zhou, Wang, He, Xiao, Hu and Lu.
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% (p = 0.002), respectively, and the disease-free survival (DFS) rates were 96.1% and 42.9% (p < 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% (p = 0.40) and DFS rates of 93.3% and 100% (p = 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.© 2023 Xie, Liu, Zhou, Wang, He, Xiao, Hu and Lu.