目前的生物学研究结果为驱动儿科患者低级别胶质瘤生长的遗传学提供了新的见解。这为新疗法提供了新靶点。本文的目的是回顾过去 24 个月内发表的儿童低级别神经胶质瘤的新疗法。低级别神经胶质瘤通常由丝裂原激活蛋白激酶 (MAPK) 改变驱动，无论是 BRAF V600E 点突变还是 BRAF融合。目前的进展还突出了成纤维细胞生长因子受体 (FGFR)、成髓细胞增多症转录因子家族 (MYB)、脑膜瘤 1 肿瘤抑制因子 (MN1)、神经营养性受体激酶受体家族 (NTRK)、Kristen RAS（大鼠肉瘤病毒）的新型融合哺乳动物致癌基因同源物 (KRAS)、受体酪氨酸激酶 ROS 原癌基因 1 (ROS1)、蛋白激酶 C α (PRKCA) 和血小板衍生生长因子受体 (PDGFR) 扩增。新疗法已被采用，并在儿科低级别神经胶质瘤中显示出令人鼓舞的结果。目前正在进行新一代泛 RAF 抑制剂和丝裂原激活蛋白激酶 - 激酶 (MEK) 抑制剂的试验。其他早期临床试验提供了针对 FGFR 融合、NTRK 融合、PDGFR 扩增和 ROS1 突变的儿科患者的安全性数据。儿科低级别胶质瘤的历史治疗方案采用手术、放射治疗和常规化疗。最近对其生物学的深入了解发现，MAPK 驱动途径的改变通常是肿瘤发生的标志。针对这些新途径，无需使用常规化疗即可控制和缩小肿瘤。然而，应谨慎行事，因为这些治疗方案仍然新颖，而且我们尚未完全了解其长期效果。尽管如此，靶向药物的新时代已经到来。版权所有 © 2024 Wolters Kluwer Health, Inc. 保留所有权利。

Current biological findings provide new insights into the genetics driving growth of low-grade gliomas in pediatric patients. This has provided new targets for novel therapies. The purpose of this paper is to review novel therapies for pediatric low-grade gliomas that have been published in the past 24 months.Low-grade gliomas are often driven by mitogen activated protein kinase (MAPK) alterations either with BRAF V600E point mutations or BRAF fusions. Current advances have also highlighted novel fusions of fibroblast growth factor receptor (FGFR), myeloblastosis family of transcription factors (MYB), meningioma 1 tumor suppressor (MN1), neurotrophic receptor kinase family of receptors (NTRK), Kristen RAS (Rat Sarcoma Virus) oncogene homolog in mammals (KRAS), Receptor tyrosine kinase ROS proto oncogene 1 (ROS1), protein kinase C alpha (PRKCA), and platelet derive growth factor receptor (PDGFR) amplification. Novel therapies have been employed and are showing encouraging results in pediatric low-grade gliomas. Current trials are underway with newer generation pan RAF inhibitors and mitogen activated protein kinase - kinase (MEK) inhibitors. Other early phase clinical trials have provided safety data in pediatric patients targeting FGFR fusion, NTRK fusion, PDGFR amplification and ROS1 mutations.Historical treatment options in pediatric low-grade gliomas have utilized surgery, radiation therapy and conventional chemotherapy. Recently greater insight into their biology has found that alterations in MAPK driven pathways are often the hallmark of tumorigenesis. Targeting these novel pathways has led to tumor control and shrinkage without the use of conventional chemotherapy. Caution should be taken however, since these treatment options are still novel, and we do not fully appreciate the long-term effects. Nonetheless a new era of targeted medicine is here.Copyright © 2024 Wolters Kluwer Health, Inc. All rights reserved.