Abstract: Objective The purpose of this study was to evaluate efficacy of preoperative neoadjuvant chemotherapy followed by high-intensity focused ultrasound (HIFU) ablation in the treatment of children with hepatoblastoma (HB). Methods 26 children with HB were encountered in our hospital between January 2015 and June 2022, neoadjuvant chemotherapy were conducted in 14 children as for control, and HIFU after neoadjuvant chemotherapy were performed in another 12 children for observation. All children were followed-up for two years. Results There were no significant differences as respect to baseline demography, diameters and locations of tumors and serum alpha-fetoprotein levels between the two groups(P>0.05); complete remission, partial remission, stable disease, objective remission rate (ORR) and disease control rate (DCR) in the observation were50.0%, 25.0%, 16.7%, 75.0% and 91.7%, while they were 14.3%, 28.6%, 35.7%, 42.8% and 78.6% in the control, only ORR significantly different between the two groups (P<0.05); during period of antitumor therapy, there were no significant differences as respect to incidences of adverse effects, such as alopecia, nausea and vomiting, granulocytopenia, thrombocytopenia, hemoglobin reduction and sinus tachycardia between the two groups (P>0.05); by end of two-year follow-up, the mortality rates in the observation group was 25.0%, not significantly different as compared to 35.7% in the control group (P>0.05). Conclusion The strategy of HIFU ablation after neoadjuvant chemotherapy in treatment of children with HB is efficacious, which might improve short-term remission, while the long-term survival benefit needs further observation.

Key words: Hepatoblastoma, Neoadjuvant chemotherapy, High-intensity focused ultrasound, Ablation, Therapy