Excellent Outcomes With High-Risk Pediatric Hepatoblastoma: A Detailed Analysis of a Large, Single-Center Experience

Hepatoblastoma (HBA) is a rare pediatric malignancy treatable with liver transplantation (LTx). Limited data exist on outcomes after LTx for HBA. This study evaluates the therapeutic efficacy of LTx in pediatric patients with HBA, delineates principal complications, and assesses long-term psychosocial outcomes.

This retrospective cohort study analyzed a prospectively maintained database of children undergoing LTx for HBA at a single US center from 1984 to 2025. Primary outcomes were patient and graft survival at 1, 5, and 10 years post-LTx and HBA recurrence. Secondary outcomes included post-LTx complications, recurrence predictors, and psychosocial parameters.

Of 55 patients (60% female; 40% Hispanic; mean age at diagnosis, 36 months), most had PRETEXT stage III (44%) or IV (47%) disease; mean α-fetoprotein level at diagnosis was 569 355 ng/mL. All but 1 received chemotherapy; 15 (27%) had pre-LTx resection. Mean wait time to LTx was 10 months. Pre-LTx metastatic disease (pulmonary) was present in 5 patients (9%). Explant pathology showed macrovascular invasion (n = 16), positive lymph nodes (n = 3), and extra-hepatic tumor invasion/rupture (n = 2). Post-LTx complications included HBA recurrence (20%), hepatic artery thrombosis (11%), and biliary stricture (20%). Patient survival was 89% at 1 year, 74% at 5 and 10 years; graft survival was 89%, 73%, and 73%, respectively. Significant predictors of recurrence were older age at LTx (p = 0.01), longer waiting time to LTx (p = 0.02), prior resection (p = 0.01), larger tumor size (p = 0.03), vascular invasion (p = 0.02), and utilization of a segmental graft (p = 0.002). Mortality was mainly due to recurrence. Psychosocially, 14 patients needed special education, 10 had developmental delays, 5 of 12 adults completed high school, 2 earned college degrees, and 3 are employed.

This large single-center study demonstrates favorable survival after LTx for high-risk HBA. Recurrence predictors support avoiding pre-LTx resection in high-risk cases. While living donor or split grafts can expedite transplantation, segmental grafts were independently linked to higher recurrence. Notably, metastases at diagnosis did not increase recurrence, supporting aggressive treatment. The rate of HAT and biliary stricture was significantly higher for children transplanted for HBA than for our overall pediatric experience. These findings help guide the management of this rare malignancy.