Hepatocellular carcinoma in children and adolescents: results of a retrospective analysis

Hepatocellular carcinoma (HCC) is a rare primary liver malignancy in children and adolescents with poor prognosis.

The aim of the study – to evaluate the clinical characteristics, treatment strategies, and outcomes in pediatric and adolescent patients with HCC, and to identify factors influencing survival.

Materials and methods. A retrospective analysis was conducted on 39 patients aged 4 to 16 years with histologically confirmed HCC treated and consulted at the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology between 2012 and 2023. Demographic and clinical features, underlying predisposing conditions, laboratory findings, intrahepatic tumor spread (PRETEXT staging), surgical interventions, and systemic treatment regimens were analyzed.

Results. Median age was 133.5 months (11.1 years; range 49.7–199.4 months). Male to female ratio was 1.1:1. HCC developed de novo in 77 % of patients, while 23 % had predisposing conditions – most commonly chronic hepatitis B. The clinical presentation was predominantly non-specific, and in 28.9 % of cases, the tumor was discovered incidentally. Serum alpha-fetoprotein levels were within normal limits in 45% of patients, particularly in those with fibrolamellar HCC.

Advanced intrahepatic tumor spread (PRETEXT stage III–IV) was observed in 60 % of cases, and distant metastases were present in 18 % of patients. The 5-year overall survival (OS) was 37 %, and the event-free survival was 26 %. The presence of distant metastases was significantly associated with inferior outcomes (5-year OS: 45 % in non-metastatic vs. 0 % in metastatic cases, p < 0.001). PRETEXT stage and histological subtype showed no significant impact on OS or event-free survival.

Surgical treatment (hepatic resection or orthotopic liver transplantation) was performed in 66 % of patients and was significantly associated with improved outcomes: 5-year OS was 54 % in the surgical group versus 0 % in those who did not undergo surgery (p < 0.001). Liver transplantation showed a trend toward better outcomes compared to resection, although the difference was not statistically significant. Initial treatment strategy (primary resection vs. biopsy with neoadjuvant chemotherapy) had no impact on survival.

Conclusions. Pediatric HCC is a rare but highly aggressive malignancy that is often diagnosed at an advanced stage. Radical surgical resection, including liver transplantation in certain cases, remains the primary determinant of prognosis. Improved outcomes require early diagnosis, early discussion of therapeutic tactics in center specialized in hepatobiliary surgery and inclusion of pediatric patients in multicenter clinical trials.

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