用氨酸激酶抑制剂治疗的固体瘤儿科患者的分子预选择
Peter Sisovsky1,2, Nikola Gejgusova1, Tomas Fazekas3
1Efficacy Assessment Department, State Institute for Drug Control, Bratislava, Slovakia.
Future oncology (London, England)
|January 29, 2026
概括
分子预选择显著改善了儿科固体瘤中氨酸激酶抑制剂 (TKI) 反应率. 目前用于TKI治疗的患者选择方法需要加强,以获得更好的结果.
科学领域:
- 在瘤学瘤学.
- 药物基因组学 药物基因组学
- 儿科医学 儿科医学
背景情况:
- 氨酸激酶抑制剂 (TKIs) 在治疗儿科固体瘤方面至关重要.
- 优化TKI疗效需要了解分子驱动因素和患者选择.
- 目前的治疗策略可能无法充分利用分子洞察力.
研究的目的:
- 审查分子预选择和组合治疗对儿童非中枢神经系统 (CNS) 固体瘤的整体反应率 (ORR) 的影响.
- 评估分子引导TKI治疗与标准方法的有效性.
- 确定TKI治疗的分子预选择标准需要改进的领域.
主要方法:
- 临床研究的系统性文献综述报告了儿童固体瘤TKI治疗的ORR (直到2023年7月).
- 分析涉及分子预选患者,TKI单一治疗和组合治疗的研究.
- 使用MINORS评分对方法质量的评估.
主要成果:
- 分子预选择显著增加了TKI单疗中的中位数ORR,从0%增加到36% (p=0.001).
- 在分子未选择的患者中,组合治疗改善了中位数ORR至14% (p=0.003).
- 方法质量中等到差;大多数研究都缺乏全面的分子表征.
结论:
- 瘤分子背景对于在儿科固体瘤中有效使用TKI至关重要.
- 目前对技术知识产权的分子预选标准是不够的,需要改进.
- 需要进一步的研究,以开发更好的分子分析和选择策略,用于TKI治疗.
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