Treatment and outcomes of hepatoblastoma in a tertiary care pediatric institution: a single-center experience

Vollständiger Abstract

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Introduction Hepatoblastoma is the most prevalent pediatric malignant liver neoplasm, and despite substantial advances in multimodal treatment, outcome data from Southeast European pediatric centers remain limited. The aim of this study was to evaluate the clinical characteristics, treatment approaches, surgical management, and outcomes of children treated for hepatoblastoma at a tertiary pediatric institution over a 17-year period. Methods This retrospective single-center cohort study included 24 pediatric patients treated between January 2008 and March 2025. Clinical variables included age, sex, tumor size, PRETEXT stage, metastatic disease, and vascular involvement. Treatment variables comprised chemotherapy protocol, surgical procedure, liver transplantation, and resection margin status, while outcome measures included relapse, overall survival, and normalization of serum α-fetoprotein levels. Results With the exception of one child who died before treatment could be initiated, all patients underwent surgical treatment following liver biopsy and preoperative chemotherapy. Overall, six children (25.0%) died, whereas 18 were alive at the time of analysis, corresponding to an overall survival rate of 75.0% with a median follow-up of 9.0 years. All patients with PRETEXT 1 and PRETEXT 2 disease survived, whereas survival among patients with PRETEXT 3–4 disease was significantly lower (100.0% vs. 50.0%, p = 0.014). Postoperative relapse occurred in two of the 23 treated patients (8.7%), and serum α-fetoprotein levels normalized after treatment in all surviving patients. Discussion In this retrospective single-center cohort, multimodal treatment of hepatoblastoma achieved an overall survival of 75.0%, with particularly favorable outcomes in patients with PRETEXT 1–2 disease and negative surgical margins. These findings provide valuable long-term real-world evidence on hepatoblastoma management at a tertiary pediatric referral center.