目的 探讨小分子靶向药物对于儿童进展低级别胶质瘤（pediatric low-grade gliomas, pLGG）的疗效。 方法 回顾性分析首都医科大学附属北京世纪坛医院儿科收治的自2021年7月开始口服小分子靶向药物的pLGG患儿，对其疗效及安全性进行分析。 结果 共20例患儿纳入研究。曲美替尼组12例（其中BRAF融合11例，BRAF V600E突变1例），4例部分缓解（33%），2例微小缓解（17%），中位起效时间3.0个月。维莫非尼组6例（BRAF V600E突变6例），5例部分缓解（83%），中位起效时间1.0个月。维莫非尼组与曲美替尼组两组无进展生存率比较差异无统计学意义（P>0.05），两组中位疗效持续时间（部分缓解+微小缓解+疾病稳定）分别为11.0个月和18.0个月。奥拉帕利组1例（ATM突变）与依维莫司组1例（NF1突变）分别应用24和21个月，因疾病稳定停药。各组均未监测到严重不良反应。 结论 小分子靶向药物用于pLGG时，可使患儿获益且耐受性良好；BRAF V600E突变患儿使用维莫非尼治疗缓解率高，且早期出现肿瘤缩小，可尝试用于初治pLGG患儿。

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.