Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong SAR, China.
School of Pharmacy & Medical Sciences, University of Bradford, Bradford BD7 1DP, UK.
Future Oncol. 2017 Jun;13(15):1345-1358. doi: 10.2217/fon-2017-0003. Epub 2017 Jun 7.
Currently, out of the 82 US FDA-approved targeted therapies for adult cancer treatments, only three are approved for use in children irrespective of their genomic status. Apart from leukemia, only a handful of genomic-based trials involving children with solid tumors are ongoing. Emerging genomic data for pediatric solid tumors may facilitate the development of precision medicine in pediatric patients. Here, we provide an up-to-date review of all reported genomic aberrations in the eight most common pediatric solid tumors with whole-exome sequencing or whole-genome sequencing data (from cBioPortal database, Pediatric Cancer Genome Project, Therapeutically Applicable Research to Generate Effective Treatments) and additional non-whole-exome sequencing studies. Potential druggable events are highlighted and discussed so as to facilitate preclinical and clinical research in this area.
目前,美国食品和药物管理局批准的 82 种成人癌症治疗靶向疗法中,只有 3 种获准不分基因组状况用于儿童。除白血病外,只有少数涉及儿童实体瘤的基于基因组的试验正在进行中。儿科实体瘤的新兴基因组数据可能有助于在儿科患者中开发精准医疗。在这里,我们提供了一份最新的综述,介绍了所有报道的 8 种最常见的儿科实体肿瘤的基因组异常,这些肿瘤有全外显子组测序或全基因组测序数据(来自 cBioPortal 数据库、儿科癌症基因组计划、治疗性应用研究以产生有效治疗)和其他非全外显子组测序研究。突出显示并讨论了潜在的可用药事件,以促进该领域的临床前和临床研究。