我们的研究旨在探索患有神经营养性原肌球蛋白受体激酶 (NTRK) 融合肿瘤的儿童和青少年的真实治疗方案，强调治疗途径、反应、副作用和结果。汇集了 17 个儿科病例（11 个软组织肉瘤）的临床数据，五种脑肿瘤和一种神经母细胞瘤）接受拉罗替尼治疗，并对 14 名患者的放射学图像进行了集中审查。对治疗反应不佳、肿瘤进展或侵袭性促使进行基因融合测试。检测到六种不同的 NTRK 融合亚型，并报告了测试和药物的各种支付来源。放射学检查显示 14 名患者中有 11 名出现客观肿瘤缓解 (OR)： 完全缓解：2 例；部分答复：九；病情稳定：3例。不良事件 1 级或 2 级通用术语标准 5 名患者报告了不良反应。关于整个队列的临床信息，17 名患者中有 15 名仍然存活（中位观察时间：25 个月）：4 名没有疾病证据，11 名患有疾病（10 名没有进展）。一名患者对 NTRK 抑制剂产生耐药性并死于疾病进展，而另一名患者则因不相关的原因死亡。这项真实世界的研究证实了拉罗替尼在患有 NTRK 融合肿瘤的儿童中具有良好的不可知肿瘤 OR 率。更好地协调以促进获得药物仍然是一个挑战，特别是在巴西等中等收入国家。

Our study aimed to explore real-world treatment scenarios for children and adolescents with neurotrophic tropomyosin receptor kinase (NTRK)-fused tumors, emphasizing access, responses, side effects, and outcomes.Pooled clinical data from 17 pediatric cases (11 soft-tissue sarcomas, five brain tumors, and one neuroblastoma) treated with larotrectinib and radiologic images for 14 patients were centrally reviewed. Testing for gene fusions was prompted by poor response to treatment, tumor progression, or aggressiveness.Six different NTRK fusion subtypes were detected, and various payment sources for testing and medication were reported. Radiologic review revealed objective tumor responses (OR) in 11 of 14 patients: Complete responses: two; partial responses: nine; and stable disease: three cases. Grades 1 or 2 Common Terminology Criteria for Adverse Events adverse effects were reported in five patients. Regarding the entire cohort's clinical information, 15 of 17 patients remain alive (median observation time: 25 months): four with no evidence of disease and 11 alive with disease (10 without progression). One patient developed resistance to the NTRK inhibitor and died from disease progression while another patient died due to an unrelated cause.This real-world study confirms favorable agnostic tumor OR rates to larotrectinib in children with NTRK-fused tumors. Better coordination to facilitate access to medication remains a challenge, particularly in middle-income countries like Brazil.