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[儿童脑瘤的新兴向疗法和正在进行的临床试验]

Keita Terashima¹

¹Division of Neuro-Oncology, Children's Cancer Center, National Center for Child Health and Development.

No shinkei geka. Neurological surgery

|December 9, 2025

概括

精密瘤学正在通过针对性疗法,如BRAF抑制剂和NTRK抑制剂,彻底改变儿科脑瘤治疗. 这些进展为患有特定遗传变异的年轻患者提供了新的希望和更好的结果.

科学领域:

儿科神经瘤学儿科神经瘤学
分子诊断学分子诊断学
精准医学是一门精准的医学.

背景情况:

分子分析显著提升了儿科脑瘤管理.
针对性疗法现在以特定可操作的改变为指导,如BRAF V600E突变,NTRK融合,NF1通路激活和H3 K27M突变.

研究的目的:

审查目前针对儿科大脑瘤的向治疗方法.
概述将精确瘤学整合到儿科神经瘤学中的实际考虑,特别是在日本.

主要方法:

审查当前的向疗法和正在进行的研究.
讨论分子分析及其在治疗选择中的作用.
强调高质量的活检对分子诊断的重要性.

主要成果:

达布拉费尼布加上特拉美丁尼布在儿科低度质瘤中显示出优越性.
拉罗特雷克提尼布和恩特雷克提尼布为NTRK融合阳性瘤提供瘤无关治疗.
塞卢梅提尼布对NF1相关的状神经纤维瘤有好处,对NF1相关的质瘤有希望.
托沃法尼布被FDA批准用于BRAF改变的瘤.
ONC201 (多达维) 已加速批准用于H3 K27M突变扩散中线质瘤.
目前,Abemaciclib正在为儿科高度质瘤和扩散中线质瘤进行评估.

结论:

以分子变化为指导的向疗法正在改变儿科脑瘤治疗.
精确瘤学为管理这些复杂疾病提供了有希望的新途径.
将精确瘤学纳入神经瘤学,重点关注日本,对于未来的进步至关重要.

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