小儿横纹肌肉瘤研究的新视角：挑战与目标。
Insights into pediatric rhabdomyosarcoma research: Challenges and goals.
机构信息
National Cancer Institute, Bethesda, Maryland.
St. Jude Children's Research Hospital, Memphis, Tennessa.
出版信息
Pediatr Blood Cancer. 2019 Oct;66(10):e27869. doi: 10.1002/pbc.27869. Epub 2019 Jun 21.
Abstract
Overall survival rates for pediatric patients with high-risk or relapsed rhabdomyosarcoma (RMS) have not improved significantly since the 1980s. Recent studies have identified a number of targetable vulnerabilities in RMS, but these discoveries have infrequently translated into clinical trials. We propose streamlining the process by which agents are selected for clinical evaluation in RMS. We believe that strong consideration should be given to the development of combination therapies that add biologically targeted agents to conventional cytotoxic drugs. One example of this type of combination is the addition of the WEE1 inhibitor AZD1775 to the conventional cytotoxic chemotherapeutics, vincristine and irinotecan.
摘要
自 20 世纪 80 年代以来，高危或复发性横纹肌肉瘤 (RMS) 儿科患者的总生存率并未显著提高。最近的研究已经确定了 RMS 中许多可靶向的弱点，但这些发现很少转化为临床试验。我们建议简化选择药物进行 RMS 临床评估的过程。我们认为，应认真考虑开发将生物靶向药物与传统细胞毒性药物联合使用的联合疗法。这种联合疗法的一个例子是将 WEE1 抑制剂 AZD1775 加入到常规细胞毒性化疗药物长春新碱和顺铂中。
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