Chemotherapeutic approaches for newly diagnosed hepatoblastoma: past, present, and future strategies

Surgical resection is the foundation of therapy in hepatoblastoma (HB), yet most patients have unresectable tumors at diagnosis. Patients with resectable tumors have event-free survival (EFS) of 80-90% and can be cured with cisplatin, 5-fluorouracil, and vincristine. Patients whose tumors are unresectable but without overt metastases at diagnosis have EFS of 60-70%, and many can be rendered resectable without doxorubicin. Children with metastatic disease have fared poorly with 20-50% EFS, and new approaches for these patients remain desperately needed. Dose intensification of cisplatin and doxorubicin appears beneficial in high-risk patients. Future treatment strategies, which may be useful, include increasing intensity and/or duration of therapy, developing a maintenance regimen (oral irinotecan), using liver transplantation more often for patients to undergo complete resection, and identifying and incorporating novel agents. A better understanding of the biologic and pathologic factors is critical for predicting tumor behavior and developing more logical risk-based treatments.