Children's National Hospital.
Hackensack Meridian Health.
Neoplasia. 2023 May;39:100895. doi: 10.1016/j.neo.2023.100895. Epub 2023 Mar 20.
Pediatric intracranial ependymoma has seen a recent exponential expansion of biological findings, rapidly dividing the diagnosis into several subgroups, each with specific molecular and clinical characteristics. While such subdivision may complicate clinical conclusions from historical trials, this knowledge also provides an opportunity for interrogating the major clinical and biological questions preventing near-term translation into effective therapy for children with ependymoma. In this article, we briefly review some of the most critical clinical questions facing both patient management and the construct of future trials in childhood ependymoma, as well as explore some of the current barriers to efficient translation of preclinical discovery to the clinic.
小儿颅内室管膜瘤的生物学研究成果近年来呈指数级增长,迅速将诊断分为几个亚组,每个亚组都具有特定的分子和临床特征。虽然这种细分可能会使历史试验的临床结论复杂化,但这些知识也为研究主要的临床和生物学问题提供了机会,这些问题阻碍了将有效疗法在短期内应用于患有室管膜瘤的儿童。在本文中,我们简要回顾了小儿室管膜瘤在患者管理和未来临床试验构建方面面临的一些最关键的临床问题,并探讨了将临床前发现高效转化为临床实践的一些当前障碍。