文章：

从近期对年轻ATRT患者的多模式治疗管理中我们学到了什么？

What Have We Learnt from the Recent Multimodal Managements of Young Patients with ATRT?

原文发布日期：26 March 2025

DOI: 10.3390/cancers17071116

类型: Article

开放获取: 是

英文摘要：

Atypical teratoid rhabdoid tumors are rare embryonal tumors of the nervous system mainly seen in very young children with aggressive behavior and dismal prognosis when treated with conventional chemotherapy only. More recent multimodal strategies combining, variably, high dose chemotherapy, radiotherapy and or intrathecal chemotherapy have led to some stride in survival. We present the results of the most recent clinical trials and registry data for patients treated with these multimodal approaches with survival ranging from 37.1% to 88.9%. We review the current consensus of the molecular characterization of these tumors into 3 subgroups (ATRT-TYR, ATRT-SHH and ATRT-MYC) and discuss the potential clinical impact of molecular subgrouping on survival. We explore other therapeutic tools including intrathecal chemotherapy and maintenance and possible new targeted agents for patients failing multimodal strategies

摘要翻译： 

非典型畸胎样横纹肌样瘤是一种罕见的神经系统胚胎性肿瘤，主要见于低龄儿童，具有侵袭性生物学行为。若仅采用传统化疗方案治疗，其预后极差。近年来，通过联合大剂量化疗、放疗和/或鞘内化疗等多模式治疗策略，患者生存率取得了一定进展。本文汇总了采用多模式治疗方案的最新临床试验及注册数据，患者生存率介于37.1%至88.9%之间。我们回顾了当前将此类肿瘤分子特征分为三个亚型（ATRT-TYR、ATRT-SHH和ATRT-MYC）的共识，并探讨了分子分型对生存预后的潜在临床意义。同时，针对多模式治疗失败的患者，我们探讨了鞘内化疗、维持治疗等其他治疗手段，以及潜在的新型靶向药物应用前景。

原文链接：

What Have We Learnt from the Recent Multimodal Managements of Young Patients with ATRT?