Clinical Characteristics and Outcomes of Neonatal Hepatoblastoma: A Single Center Study.

OBJECTIVE

Hepatoblastoma (HB) diagnosed within one month following birth qualifies for a diagnosis of neonatal HB, whose prognosis is reportedly controversial, and its treatment is challenging. This study discussed the diagnosis, treatment, and outcomes of neonatal HB at a single center so as to enhance its overall management in the future.

METHODS

The clinical information of babies diagnosed with neonatal HB at our center from August 2009 to September 2023 were retrospectively analyzed for demographics, clinical features, therapy, and outcomes. The outcomes were estimated by the Kaplan-Meier analysis method.

RESULTS

The study comprised 79 patients aged less than one year old, among which 14 had neonatal HB whereas 65 were non-neonatal HB patients. No differences were found between groups regarding gender, birth weight, delivery details, parental age, clinical signs, or treatment strategies. Neonatal HB patients were more likely to have PRETEXT I-II, smaller tumor size, congenital diseases, and lower risk tumor grade (p < 0.05). Additionally, the AFP levels of all neonatal HB patients were greater than 10,000 ng/ml (p = 0.009) and they had higher levels of ferritin (p = 0.003) and hemoglobin (p = 0.021), but lower levels of serum total proteins (p = 0.001). The three-year survival rate (100% vs 90.8%) and three-year event-free survival rate (100% vs 86.2%) in the neonatal HB group were higher than the non-neonatal HB group.

CONCLUSION

Neonatal HB patients have unique clinical features and can achieve an excellent prognosis following combined treatment with surgery and chemotherapy. Tumor resection, when carefully performed, was safe even in babies younger than one months old. Further and long-term studies are needed from a larger neonatal HB population.

LEVEL OF EVIDENCE

Level III.