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针对性DNA测序用于在患有脑外固体瘤的儿童中量身定制的治疗方法

Nataliya A Andreeva¹, Tatyana V Shamanskaya¹, Denis Y Kachanov¹

¹Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow 117198, Russia.

International journal of molecular sciences

|December 11, 2025

概括

下一代测序在39.5%的儿科固体瘤中确定了向治疗 (TT) 预测因素. 在6.9%的患者中,TT显示出有希望的疗效和良好的耐受性,这表明了更新的治疗标准.

科学领域:

儿科瘤学儿科瘤学
分子诊断学分子诊断
癌症基因组学癌症基因组学

背景情况:

分子向治疗 (TT) 对儿科瘤学至关重要,通常以下一代测序 (NGS) 为指导.
TT在儿科癌症中很少使用,通常在瘤不可知的基础上使用.
识别预测性生物标志物对于优化年轻患者的TT疗效至关重要.

研究的目的:

调查NGS在儿童脑外固体瘤中识别TT候选者的实用性.
评估TT在这个患者队列中的疗效,持续时间和耐受性.
评估与TT相关的临床益处和无进展生存率.

主要方法:

分析了304名患有脑外固体瘤的儿科患者队列.
使用定制面板对关键癌症相关基因进行了瘤DNA测序.
下一代测序检测到核酸替代,小插入/删除和副本数量的改变.

主要成果:

在39.5%的患者 (120/304) 中发现了TT敏感性预测因子.
针对性治疗在6.9%的病例中开始 (21/304),通常作为一线或第一次复发治疗.
观察到的临床益处率为66.7%,中位数TT持续时间为10.9-12.3个月,耐受性良好.

结论:

在儿童固体瘤中,NGS有效地识别了TT候选人.
向治疗在儿科瘤学中显示出有希望的疗效和良好的耐受性.
这些发现支持更新基于分子分析的特定儿科癌症的治疗标准.

关键词:

在DNA测序过程中,DNA测序没有NGS,没有NGS.孩子们的孩子们的孩子们的孩子们分子疗法的分子疗法.神经母细胞瘤的神经母细胞瘤精密处理精密处理有针对性的治疗.

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