Introduction: Survival of patients with hepatocellular carcinoma (HCC) without recurrence depends on complete surgical resection. Determining the optimal surgical approach for pediatric patients with HCC involves a critical decision between surgical resection and liver transplantation (LT). LT is performed in patients with unresectable HCC and tumors developed on the background of a pre-existing liver disease. The optimal criteria for selecting pediatric patients with HCC for LT are not clearly established, reflecting the rarity and heterogeneity of the disease in this population.
Methods: The aim of this multicenter, observational, retrospective cohort study conducted within the TransplantChild European Reference Network was to assess  long-term results of LT in pediatric patients with HCC. In the analysis were included 72 patients < 18 years old who underwent LT for HCC between January 1, 2000, and December 31, 2022. Patients with incidentally found HCC in the explanted liver were excluded. We evaluated the effect of different factors on outcomes using the Kaplan–Meier method and log-rank test.
Results: The median age at diagnosis was 100 months (range: 3 to 213.5). In 43 (59.7%) HCC was associated with pre-existing liver disease. In 62 (13.9%) primary LT was performed, 10 (15.4%) underwent rescue LT. 24 (33.4%) were classified as PRETEXT IV, 16 (22.2%) were PRETEXT III, 17 (23.6%) were PRETEXT II, and 15 (20.8%) were PRETEXT I. A multifocal tumour was found in 50 (69.4%). 45 (62.5%) underwent LT beyond Milan criteria. The median age at LT was 105.2 months (range: 6 to 214.8). In 20 (27.8%) living donor LT was performed. 32 (44.4%) received preoperative chemotherapy, and 30 (41.7%) received adjuvant chemotherapy. In 7 (9.7%) fibrolamellar HCC was found. The median follow-up time was 70.25 months (0 to 278). Tumor recurrence was observed in 11 (15.3%), 10 of them died. 3 patients died due to transplant related causes.  Estimated patient survival after 1, 5, and 10 years was 93%, 84%, and 76%. There was no patients survival difference between patients with and without underlying disease (p=0.83). No difference was observed between children transplanted within vs. beyond Milan criteria (p=0.868). Estimated patient survival 10 years after living donor LT and deceased donor LT  was similar (p = 0.8967). Estimated patient survival after 1, 5, and 10 years was 95%, 89%, and 83% after primary LT and 78%, 56%, and 37% after rescue LT, respectively, and difference was statistically significant (p=0.00442).
Conclusion: LT in children with HCC demonstrates excellent clinical outcome, with high patient survival rates over long time even in patients outside of adult Milan criteria.  Survival analyses demonstrated a superior long-term survival of children after primary LT when compared to children after rescue LT. Presence of underlying disease, and donor type did not affect the long-term outcome of patients after LT due to HCC.