儿科非典型畸胎瘤/横纹肌瘤的分子靶向疗法。

IF 1.9 4区医学 Q2 PEDIATRICS

Pediatric Investigation Pub Date : 2022-05-23 eCollection Date: 2022-06-01 DOI:10.1002/ped4.12325

Chang Zhang, Hao Li

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引用次数: 0

摘要

非典型畸形/横纹肌瘤（AT/RTs）是一种致命的中枢神经系统肿瘤，主要在婴儿中确诊。目前治疗 AT/RT 的方法包括手术、放疗和化疗；这些治疗方法的预后较差，副作用大。AT/RT 发病机制的关键基因事件是 SMARCB1 或 SMARCA4 失活。最近的表观遗传学研究表明，相互的和亚型特异性的表观遗传学失调推动了肿瘤的发生；利用这些潜在的靶点可能会改善 AT/RT 令人沮丧的治疗结果。本综述旨在总结有关儿科 AT/RTs 靶向分子疗法的文献。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Molecular targeted therapies for pediatric atypical teratoid/rhabdoid tumors.

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Molecular targeted therapies for pediatric atypical teratoid/rhabdoid tumors.

Atypical teratoid/rhabdoid tumors (AT/RTs) are lethal central nervous system tumors, which are primarily diagnosed in infants. Current treatments for AT/RTs include surgery, radiotherapy, and chemotherapy; these treatments have poor prognoses and challenging side effects. The pivotal genetic event in AT/RT pathogenesis comprises the inactivation of SMARCB1 or SMARCA4. Recent epigenetic studies have demonstrated mutual and subtype-specific epigenetic derangements that drive tumorigenesis; the exploitation of these potential targets might improve the dismal treatment outcomes of AT/RTs. This review aims to summarize the literature concerning targeted molecular therapies for pediatric AT/RTs.

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