目的 评估剂量增强型C5VD（顺铂+5-氟尿嘧啶+长春新碱+多柔比星）方案治疗儿童局部晚期肝母细胞瘤（hepatoblastoma, HB）的初步疗效和安全性。 方法 前瞻性纳入2020年1月—2023年12月上海交通大学医学院附属上海儿童医学中心和上海市儿童医院采用剂量增强型C5VD方案治疗的24例初治儿童局部晚期HB患儿为研究对象，分析其临床特征、疗效和化疗毒性。 结果 共纳入24例HB患儿，其中男性13例，女性11例，中位诊断年龄为18.7（范围：3.5~79.4）个月。24例肝脏病灶均手术肉眼完全切除，未进行肝移植。2个疗程化疗后血清甲胎蛋白明显下降。中位随访时间为38.4（范围：15.8~50.7）个月，24例持续完全缓解，3年无事件生存率和总生存率均为100%。在144个总疗程中，3~4级中性粒细胞减少、血小板减少和感染的发生率分别为97%、77%和71%，无治疗相关死亡事件发生。5例（21%）发生了Brock 3级及以上严重听力损害，其中1例需要佩戴助听器。 结论 剂量增强型C5VD方案治疗初治儿童局部晚期HB疗效显著。化疗毒性主要表现为3~4级骨髓抑制和感染，整体安全性良好。然而，需要特别关注高剂量顺铂化疗引起的耳毒性，并且进一步优化耳保护策略。

Objective To assess the preliminary efficacy and safety of a dose-intensified C5VD regimen (cisplatin, 5-fluorouracil, vincristine, and doxorubicin) in children with locally advanced hepatoblastoma. Methods This prospective study enrolled 24 children with newly diagnosed, locally advanced hepatoblastoma who received the dose-intensified C5VD regimen at Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine, and Shanghai Children's Hospital between January 2020 and December 2023. Clinical characteristics, treatment outcomes, and chemotherapy-related toxicities were analyzed. Results Of the 24 patients, 13 were male and 11 were female, with a median age at diagnosis of 18.7 months (range: 3.5-79.4 months). All patients achieved complete macroscopic resection of hepatic lesions without liver transplantation. Serum alpha-fetoprotein levels decreased significantly after two chemotherapy cycles. During a median follow-up of 38.4 months (range: 15.8-50.7 months), all patients maintained continuous complete remission, with 3-year event-free survival and overall survival rates of 100%. Across 144 chemotherapy cycles, the incidence rates of grade 3-4 neutropenia, thrombocytopenia, and infections were 97%, 77%, and 71%, respectively; no treatment-related deaths occurred. Notably, 5 patients (21%) developed Brock grade ≥3 hearing loss, of whom 1 required a hearing aid. Conclusions The dose-intensified C5VD regimen demonstrates significant efficacy with an overall favorable safety profile in the treatment of newly diagnosed, locally advanced pediatric hepatoblastoma. Grade 3-4 myelosuppression and infection are the predominant toxicities. However, high‑dose cisplatin-induced ototoxicity remains a concern, highlighting the need for improved otoprotective strategies.