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期刊来源：Nat Med

原文链接：https://doi.org/10.1038/s41591-024-03044-0

摘要内容如下：

最近的研究表明，精准医疗可以为儿童癌症患者确定新的治疗策略。然而，目前尚不清楚哪些患者将从精确引导治疗（PGT）中获益最多。在此，我们报告了384名高危儿童癌症患者（预期治愈率低于30%）的连续数据，这些患者在零儿童癌症精准医疗计划（PRISM）儿童癌症精准医疗试验中至少随访了18个月。共有256名（67%）患者接受了PGT建议，110名（29%）患者接受了推荐治疗。与标准治疗相比，PGT的客观缓解率为36%，2年无进展生存期有所改善（26%对12%；p=0.049）或不受分子发现指导的靶向药物（26%对5.2%；P=0.00 3）。PGT基于Tier 1证据，PGT靶向融合或在疾病进展前开始具有最大的临床益处。我们的数据显示，通过全面的分子分析，PGT显著改善了患有高危癌症的儿童的预后。ClinicalTrials.gov注册：NCT03336931。

英文原文如下：

Abstracts

Recent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment (PGT). Here we report consecutive data from 384 patients with high-risk pediatric cancer (with an expected cure rate of less than 30%) who had at least 18 months of follow-up on the ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial. A total of 256 (67%) patients received PGT recommendations and 110 (29%) received a recommended treatment. PGT resulted in a 36% objective response rate and improved 2-year progression-free survival compared with standard of care (26% versus 12%; P = 0.049) or targeted agents not guided by molecular findings (26% versus 5.2%; P = 0.003). PGT based on tier 1 evidence, PGT targeting fusions or commenced before disease progression had the greatest clinical benefit. Our data show that PGT informed by comprehensive molecular profiling significantly improves outcomes for children with high-risk cancers. ClinicalTrials.gov registration: NCT03336931.

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