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Updated: Jul 23, 2025

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通过延长治疗IKZF1删除和减少其他风险组的治疗来改善ALL的结果

Rob Pieters^1,2, Hester de Groot-Kruseman^1,2, Marta Fiocco^1,2,3,4

¹Princess Máxima Center for Pediatric Oncology, Utrecht, the Netherlands.

Journal of clinical oncology : official journal of the American Society of Clinical Oncology

|July 17, 2023

概括

ALL11协议通过根据最小残留疾病调整治疗来改善急性淋巴细胞白血病 (ALL) 的儿童的结局. 长期治疗IKZF1被删除的ALL降低了复发率,而对其他组的治疗减少维持了优异的生存率.

科学领域:

儿科瘤学儿科瘤学
血液学血液学血液学
临床试验分析

背景情况:

全球性淋巴细胞白血病 (ALL) 急性淋巴细胞白血病 (ALL) 治疗的ALL10协议按最小残留疾病 (MRD) 风险组分层次.
虽然有效,ALL10在唐氏综合征 (DS) 中表现出高毒性和IKZF1-删除 (IKZF1del) ALL的不良结果,需要调整协议.

研究的目的:

评估修改后的ALL11协议在改善儿科ALL患者的治疗结果方面的有效性.
为了降低特定ALL亚型的治疗强度,包括IKZF1del,DS,ETV6::RUNX1,以及不良预尼松反应者 (PPRs),同时保持或改善生存率.

主要方法:

在ALL11协议中,招募了819名儿科ALL患者 (年龄为1-18岁),分层类似于ALL10.
治疗修改包括长时间维持IKZF1delALL,减少DS,ETV6::RUNX1和PPRALL的化疗.
结果与历史ALL10队列进行了比较.

主要成果:

通过ALL11协议实现了5年总生存率94.2%,无事件生存率为89.0%.
对IKZF1delALL的长期维持治疗显著降低了复发的累积发病率 (CIR) 的2.2倍,并改善了EFS.
对DS,ETV6::RUNX1和PPRALL的减少化疗并没有影响5年生存结果.

结论:

长期维持治疗对IKZF1delALL儿童有益,显著降低复发率.
对特定ALL亚型 (ETV6::RUNX1,DS,PPR) 的ALL11的化疗降低是成功的,但没有对患者的结果产生负面影响.
这些发现来自于与历史对照的非随机研究,表明了针对儿童ALL治疗的精细风险调整方法.

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