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在儿童中治疗耐火/复发前体B型急性淋巴细胞白血病的短期布利纳图马布

Jiao Xie¹, Suxiang Liu¹, Ming Zhou¹

¹Department of Hematology and Oncology, Children's Hospital of Soochow University, Suzhou, China.

Frontiers in pediatrics

|August 21, 2023

概括

短时间的布利纳马布治疗有效地治疗儿科耐药或复发的前体B细胞急性淋巴细胞白血病 (R/R-BCP-ALL),实现高缓解率与可控毒性. 这种方法为发展中国家提供了一个具有成本效益的选择.

科学领域:

儿科血液瘤学
免疫治疗是一种免疫疗法.
患有急性淋巴细胞白血病.

背景情况:

耐药或复发的前体B细胞急性淋巴细胞白血病 (R/R-BCP-ALL) 在儿科瘤学中提出了重大治疗挑战.
双特异性抗体BLINATUMOMAB在治疗BCP-ALL方面表现有前途,但其在儿科R/R病例中短期治疗方案的有效性需要评估.

研究的目的:

在被诊断患有R/R-BCP-ALL的儿童中评估短暂的布利纳图马布治疗疗程的临床疗效和安全性.
为了确定缓解率,总生存率 (OS) 和短期布利纳马布治疗后的无疾病生存率 (DFS).

主要方法:

对33名患有R/R-BCP-ALL的儿科患者 (0-18岁) 的临床数据的回顾性分析,这些患者接受了14天的布利纳图马布疗程治疗.
数据收集于2021年8月至2022年11月期间,重点关注缓解的实现,生存结果以及诸如细胞因子释放综合征 (CRS) 和免疫效应细胞相关神经毒性综合征 (ICANS) 等不良事件.

主要成果:

观察到78.8% (26/33) 的高完全缓解率 (CR),在耐火病例中缓解率为90.9%,在复发病例中缓解率为72.7%.
总生存率 (OS) 和无疾病生存率 (DFS) 在获得CR的患者中 (96.1%的OS,96.1%的DFS) 与没有CR的患者相比 (57.1%的OS,42.9%的DFS) 显著更高.
一般来说,不良事件是低的和可控的,包括54.5%的CRS发病率 (2个严重的病例) 和3.0%的ICANS发病率 (没有严重的病例),没有与治疗相关的死亡.

结论:

短时间的布利纳图马布疗程在儿科R/R-BCP-ALL中显示出高疗效和良好的安全性,达到相当大的缓解率.
这种治疗提供了一个潜在的成本效益和节约资源的治疗选择,特别有利于发展中国家.
桥梁造血干细胞移植 (HSCT) 在这个队列中没有显示出显著的短期生存益处.

关键词:

急性淋巴细胞白血病急性淋巴细胞白血病这就是Blinatumomabomab.孩子们的孩子们的孩子们的孩子们造血干细胞移植造血干细胞移植耐火/复发的情况短途旅行短途旅行

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