PDF(Pubmed)
Abstract：
Clinical outcomes for many childhood brain tumours remain poor, despite our increasing understanding of the underlying disease biology. Advances in molecular diagnostics have refined our ability to classify tumour types and subtypes, and efforts are underway across multiple international paediatric neuro-oncology consortia to take novel biological insights in the worst prognosis entities into innovative clinical trials. Whilst for the first time we are designing such studies on the basis of disease-specific biological data, the levels of preclincial evidence in appropriate model systems on which these trials are initiated is still widely variable. We have considered these issues between CONNECT, PNOC and ITCC-Brain, and developed a framework in which we can assess novel concepts being brought forward for possible clinical translation. Whilst not intended to be proscriptive for every possible circumstance, these criteria provide a basis for self-assessment of evidence by laboratory scientists, and a platform for discussion and rational decision-making prior to moving forward clinically.
摘要：
许多儿童脑肿瘤的临床结果仍然很差，尽管我们越来越了解潜在的疾病生物学。分子诊断的进展完善了我们对肿瘤类型和亚型进行分类的能力，多个国际儿科神经肿瘤学联盟正在努力将对预后最差实体的新生物学见解纳入创新的临床试验。虽然我们第一次根据疾病特异性生物学数据设计这样的研究，在启动这些试验的适当模型系统中,临床前证据水平仍然存在很大差异.我们已经考虑了这些问题之间的连接，PNOC和ITCC-Brain，并开发了一个框架，在该框架中，我们可以评估为可能的临床翻译而提出的新概念。虽然并不打算对每一种可能的情况进行规范，这些标准为实验室科学家自我评估证据提供了基础，以及在临床推进之前进行讨论和理性决策的平台。
