Addition of Vincristine and Irinotecan to Standard Therapy in a Patient With Refractory High-risk Hepatoblastoma Achieving Long-term Relapse-free Survival.

Children with metastatic hepatoblastoma have a poor prognosis, even with dose intensification of cisplatin and doxorubicin. Vincristine and irinotecan have demonstrated activity in high risk disease. This report describes a 3-year-old girl with metastatic hepatoblastoma with unresectable disease after 5 cycles of cisplatin, 5-fluorouracil, vincristine, and doxorubicin who had a complete response of her metastatic disease to vincristine and irinotecan (intravenous and oral forms), allowing surgical resection of her liver disease. She remains in remission 48 months since therapy completion.