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高危儿科癌症的精确指导治疗

Loretta M S Lau^1,2,3, Dong-Anh Khuong-Quang^4,5, Chelsea Mayoh^1,2

¹Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.

Nature medicine

|June 6, 2024

概括

精确引导治疗 (PGT) 显著改善了患有高风险癌症的儿童的治疗结果. 综合分子分析指导PGT,提高儿童癌症患者的生存率和治疗反应.

科学领域:

儿科瘤学儿科瘤学
精准医学是一门精准的医学.
癌症基因组学癌症基因组学

背景情况:

精准医学为儿童癌症提供了新的治疗策略.
确定最能从精确指导治疗 (PGT) 中获益的患者仍然是一个挑战.

研究的目的:

评估PGT在患有高风险癌症的儿童中的有效性.
确定与PGT的临床益处相关的因素.

主要方法:

在ZERO儿童癌症PRISM试验中,分析了384名高风险儿科癌症患者的连续数据.
评估PGT建议和接受的治疗方法.
在PGT和标准护理或非分子导向向药物之间比较结果 (客观反应率,无进展生存率).

主要成果:

256名患者 (67%) 接受了PGT建议;110名患者 (29%) 接受了推治疗.
普吉公司实现了36%的客观响应率.
与标准治疗 (26%对12%) 和非分子导向向药物 (26%对5.2%) 相比,PGT改善了两年无进展生存率.
基于一级证据的PGT,针对合并,或在进展之前启动,显示出最大的好处.

结论:

基于综合分子分析的PGT显著改善了患有高风险癌症的儿童的治疗结果.
与传统治疗和非指导向疗法相比,PGT显示出更高的疗效.

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