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 1 . 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 1 - 3 , 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. Pediatr Blood Cancer 2012; 59: 809–812. © 2012 Wiley Periodicals, Inc.