HEPATOBLASTOMA

EPIDEMIOLOGY.

Hepatoblastoma occurs predominantly in children <3 yr of age. The etiology is unknown.

Hepatoblastomas are associated with familial adenomatous polyposis. Alterations in the antigenpresenting cell (APC)/-catenin pathway have been found in most of the tumors evaluated.
Hepatoblastoma also is associated with Beckwith-Wiedemann syndrome, which can show a
similar loss of genomic imprinting of the insulin-like growth factor-2 gene. Low birthweight is
associated with increased incidence of hepatoblastoma, with the risk increasing as birthweight
decreases.
PATHOGENESIS.

Hepatoblastoma can be epithelial type, containing fetal or embryonal malignant cells (either as a
mixture or as pure elements), or the mixed type, containing mesenchymal and epithelial
elements. Pure fetal histology predicts a more favorable outcome.
CLINICAL MANIFESTATIONS.

Hepatoblastoma usually presents as a large, asymptomatic abdominal mass. It arises from the
right lobe 3 times more often than the left and usually is unifocal. As the disease progresses,
weight loss, anorexia, vomiting, and abdominal pain may ensue. Metastatic spread of
hepatoblastoma most commonly involves regional lymph nodes and the lungs.
A valuable serum tumor marker, -fetoprotein (AFP), is used in the diagnosis and monitoring of
hepatic tumors. AFP level is elevated in almost all hepatoblastomas. Bilirubin and liver enzymes
usually are normal. Anemia is common, and thrombocytosis occurs in about of patients.
Hepatitis B and C serology should be obtained but usually are negative in hepatoblastoma.
Diagnostic imaging should include plain radiographs and ultrasonography of the abdomen to
characterize the hepatic mass. Ultrasonography can differentiate malignant hepatic masses from
benign vascular lesions. Either CT or MRI is an accurate method of defining the extent of
intrahepatic tumor involvement and the potential for surgical resection. Evaluation for metastatic
disease should include CT of the chest and bone scan.
TREATMENT.

In general, the cure of malignant hepatic tumors in children depends on complete resection of the
primary tumor ( Fig. 504-1 ). As much as 85% of the liver can be resected, with hepatic
regeneration noted within 34 mo after surgery. Cisplatin in combination with vincristine and 5fluorouracil or doxorubicin is effective treatment for hepatoblastoma and increases the chances
of cure after complete surgical resection. In low-stage tumors, survival rates >90% can be
achieved with multimodal treatment, including surgery and adjuvant chemotherapy. With tumors
unresectable at diagnosis, survival rates of approximately 60% can be obtained. Metastatic
disease further reduces survival, but complete regression of disease often can be obtained with
chemotherapy and surgical resection of the primary tumor and isolated pulmonary metastatic
disease, resulting in survival rates of about 25%. Liver transplant is a viable option for

unresectable primary hepatic malignancies and results in good long-term survival. Pretransplant
medical condition is an important predictor of outcome, and thus transplant is much more
effective as the primary surgery than as salvage therapy.