Introduction and aims: Liver transplant (LT) is the only treatment for local unresectable hepatocellular carcinoma (HCC) in children. The study analyzed long-term survival of children with HCC exceeding or fulfilling Milan criteria treated with LT at our center.
Methods: Single-center retrospective cohort study of all children undergoing LT for HCC at Bambino Gesú Children’s Hospital in Rome from January 2009 to December 2024. LT was indicated because of unresectable localized tumor or concomitant underlying liver disease. Preoperative clinical and laboratory parameters, preoperative procedures, surgery, postoperative outcome, and survival were analyzed.
Results: Fifteen children underwent LT for HCC. Median age at diagnosis was 9 years (IQR 8,10). In 4 (26.7%) cases, HCC developed in healthy liver, while in 11 (73.3%) in children with an underlying liver disease. Six (40%) children received neoadjuvant chemotherapy, and 1 (6.7%) child underwent a preoperative chemo-embolization. Median time on the transplant waiting list was 20 days (IQR 40,145). Eight (53.3%) children exceeded Milan criteria. All children received grafts from deceased donors, split grafts in 8 (53.3%) cases and whole liver in 7 (46.7%). Median follow-up time was 88 months (IQR 40, 145). Two (13.3%) children developed distant metastases and eventually died. All other children are alive without disease recurrence. The 5-year overall survival rate was 86.7% and was not statistically different between children exceeding or fulfilling Milan criteria (p=0.226). Similarly, we found no differences in survival based on the presence of liver disease and the type of immunosuppression.
Conclusions: Transplanted children exceeding Milan criteria showed similar 5-year survival to children who underwent LT within Milan criteria. These results may indicate the need for reestablishment of specific criteria and guidelines for improving surgical treatment of HCC in children.