Background: High-grade glioma (HGG) is the most common primary malignant brain tumor, with peak incidence in the fifth and sixth decades of life. Although HGG is rare in children, the prognosis remains poor, with a median overall survival (OS) of less than two years. Recently, TRK inhibitors have been approved for the treatment of tumors harboring NTRK gene fusions. In this review, we analyzed data from early clinical trials investigating the use of these agents in patients with HGG. Methods: A systematic literature search was performed in the PubMed database. Studies involving patients with HGG treated with TRK inhibitors were included. We analyzed progression-free survival (PFS), 24-week disease control rate, and complete or partial radiological responses according to the Response Assessment in Neuro-Oncology (RANO) criteria. Results: Sixteen studies comprising 55 patients with HGG harboring NTRK gene fusions (19 adults and 36 children) were included. A statistically significant difference in PFS was observed between pediatric and adult patients treated with TRK inhibitors (17 vs. 8.5 months;p< 0.001). Pediatric patients also exhibited a higher rate of complete or partial radiological response compared to adults (94% vs. 57%). Discussion: Although the available evidence on TRK inhibitors in HGG is limited, the findings of this review highlight a potentially promising role for these agents, particularly in the treatment of pediatric HGGs.
背景：高级别胶质瘤（HGG）是最常见的原发性恶性脑肿瘤，其发病高峰出现在五六十岁。尽管HGG在儿童中较为罕见，但其预后仍然较差，中位总生存期（OS）不足两年。近年来，TRK抑制剂已被批准用于治疗携带NTRK基因融合的肿瘤。本综述分析了早期临床试验中关于这些药物在HGG患者中应用的数据。方法：在PubMed数据库中进行系统性文献检索，纳入涉及使用TRK抑制剂治疗的HGG患者的研究。我们根据神经肿瘤学反应评估（RANO）标准，分析了无进展生存期（PFS）、24周疾病控制率以及完全或部分影像学缓解情况。结果：共纳入16项研究，涉及55例携带NTRK基因融合的HGG患者（19例成人和36例儿童）。接受TRK抑制剂治疗的儿童与成人患者在PFS上存在统计学显著差异（17个月 vs. 8.5个月；p < 0.001）。与成人相比，儿童患者也表现出更高的完全或部分影像学缓解率（94% vs. 57%）。讨论：尽管目前关于TRK抑制剂在HGG中应用的证据有限，但本综述的结果强调了这些药物可能具有的潜在应用前景，尤其是在儿童HGG的治疗中。
肿瘤(癌症)患者之家