中国当代儿科杂志2025，Vol.27Issue(6)：682-689,8.DOI:10.7499/j.issn.1008-8830.2410012
儿童进展低级别胶质瘤小分子靶向治疗研究
Molecular targeted therapy for progressive low-grade gliomas in children
摘要
Abstract
Objective To evaluate the efficacy of molecular targeted agents in children with progressive pediatric low-grade gliomas(pLGG).Methods A retrospective analysis was conducted on pLGG patients treated with oral targeted therapies at the Department of Pediatrics,Beijing Shijitan Hospital,Capital Medical University,from July 2021.Treatment responses and safety profiles were assessed.Results Among the 20 enrolled patients,the trametinib group(n=12,including 11 cases with BRAF fusions and 1 case with BRAF V600E mutation)demonstrated 4 partial responses(33%)and 2 minor responses(17%),with a median time to response of 3.0 months.In the vemurafenib group(n=6,all with BRAF V600E mutation),5 patients achieved partial responses(83%),showing a median time to response of 1.0 month.Comparative analysis revealed no statistically significant difference in progression-free survival rates between the two treatment groups(P>0.05).The median duration of clinical benefit(defined as partial response+minor response+stable disease)was 11.0 months for vemurafenib and 18.0 months for trametinib.Two additional cases,one with ATM mutation treated with olaparib for 24 months and one with NF1 mutation receiving everolimus for 21 months,discontinued treatment due to sustained disease stability.No severe adverse events were observed in any treatment group.Conclusions Molecular targeted therapy demonstrates clinical efficacy with favorable tolerability in pLGG.Vemurafenib achieves high response rates and induces early tumor shrinkage in patients with BRAF V600E mutations,supporting its utility as a first-line therapy.关键词
胶质瘤/分子靶向治疗/疾病进展/儿童Key words
Glioma/Molecular targeted therapy/Disease progression/Child引用本文复制引用
孙艳玲,孙黎明,李苗,刘晶晶,高文超,武跃芳,万露露,任思其,杜淑旭,武万水..儿童进展低级别胶质瘤小分子靶向治疗研究[J].中国当代儿科杂志,2025,27(6):682-689,8.基金项目
北京世纪坛医院青年基金(2022-q19). （2022-q19）
