Primary liver cancers account for about 1% of pediatric cancers. Hepatoblastoma is the most common and usually occurs before the age of 4. Hepatocellular carcinoma is more common in older children and adolescents. Other tumors exist, including undifferentiated embryonal sarcoma, fibrolamellar carcinoma, rhabdoid tumors, vascular tumors, and rhabdomyosarcomas of the bile ducts.

The most common symptom is an enlarged abdomen or a mass discovered by the parents or the doctor. Some children, particularly those with hepatoblastoma, remain in good overall health for a long time.

Blood Markers

Alpha-fetoprotein, or AFP, is the primary marker for hepatoblastomas and certain hepatocellular carcinomas. Its interpretation must take age into account, as it is physiologically elevated in infants. Changes in AFP levels during treatment help assess the response. A normal AFP level does not rule out a liver tumor.

Imaging and the PRETEXT Classification

Ultrasound identifies the mass and assesses the blood vessels. A liver MRI or CT scan determines its extent within the different parts of the liver and its relationship to the hepatic veins, the portal vein, the vena cava, and the bile ducts. A chest CT scan looks for lung metastases. The PRETEXT classification describes the extent of the disease before treatment and helps plan surgery.

Biopsy

A biopsy is necessary in many situations to confirm the diagnosis and perform histological and molecular analyses. It may be avoided in a few very characteristic cases or when the tumor is removed immediately, depending on the protocol strategy and multidisciplinary discussion.

In most cases, no specific cause is identified. However, certain syndromes or conditions increase the risk of hepatoblastoma, including Beckwith-Wiedemann syndrome, hemihyperplasia, familial adenomatous polyposis, and prematurity with very low birth weight. Certain chronic metabolic or liver diseases increase the risk of hepatocellular carcinoma.

The treatment strategy depends on the exact type of tumor, the risk group, the PRETEXT stage, the presence of metastases, AFP levels, and the feasibility of complete resection. It must be determined very early on in consultation with a liver surgery team and, if necessary, a transplant team.

Treatment of Hepatoblastoma

The vast majority of patients with hepatoblastoma receive cisplatin-based chemotherapy before surgery. This phase treats distant disease and may make the tumor resectable. Surgery then removes the affected portion of the liver. Adjuvant chemotherapy is administered based on the risk group and the patient’s response. Immediate surgery remains an option for a few small tumors that are completely resectable.

Surgery and Transplantation

Surgery must preserve a functional volume of liver tissue as well as its blood supply and drainage. For a tumor confined to the liver but impossible to remove by partial hepatectomy, a transplant may offer a chance for a cure. The need for a transplant must be assessed early to avoid procedures that would compromise the transplant.

Lung Metastases

In hepatoblastoma, pulmonary metastases may resolve with chemotherapy. Persistent nodules may require complete resection prior to transplantation or as part of curative treatment. The strategy depends on their number, location, and response.

Hepatocellular carcinoma

Pediatric hepatocellular carcinoma is generally less responsive to chemotherapy than hepatoblastoma. Complete surgical resection is the primary goal. Transplantation may be considered for certain unresectable forms confined to the liver, but its indications differ from those for hepatoblastoma. For metastatic or unresectable disease, options are limited, and participation in a clinical trial is sought when appropriate.

Other Tumors

Undifferentiated embryonal sarcoma generally requires a combination of chemotherapy and surgery. Vascular, rhabdoid, biliary, or benign tumors require specific treatment strategies that should not be equated with the treatment of hepatoblastoma.

Localized tumors can be cured when complete surgical resection is possible. Metastatic forms, certain histologies, and tumors that remain unresectable despite chemotherapy are more difficult to treat. Hepatocellular carcinoma and fibrolamellar carcinoma generally have a less favorable prognosis when complete surgery is not feasible.

PHITT is a large international study focused on pediatric hepatoblastomas and hepatocellular carcinomas. It standardized risk stratification and evaluated strategies aimed at reducing toxicity in low-risk cases and intensifying treatment in high-risk cases.

Patient enrollment for PHITT has been completed. An international consensus published in June 2025 provides interim treatment guidelines pending the main results and a forthcoming prospective study. 

Follow-up care is designed to detect recurrence, monitor AFP levels when this marker was elevated, and manage late effects. It is tailored to the tumor, medications, surgery, and any potential transplant.

The Department of Pediatric and Adolescent Oncology coordinates patient care with the teams in imaging, pathology, clinical laboratory medicine, genetics, liver surgery, transplantation, and supportive care. The treatment strategy is discussed during specialized multidisciplinary team meetings.

Complex liver procedures and transplants are organized in collaboration with expert partner institutions. Early coordination helps avoid delays in surgery or enrollment in a transplant program when such a procedure is indicated.