Liver transplantation and chemotherapy in children with unresectable primary hepatic malignancies: Development of a management algorithm

OBJECTIVES: Total surgical excision and adjunctive chemotherapy are cornerstones of treatment of primary hepatic malignancies in children. Recent studies suggest that transplantation is a viable option for unresectable tumors, but there are questions concerning decision making regarding resectability and timing of transplantation in relation to chemotherapy. We developed a management algorithm based on our experience, with reference to recently published multicenter transplantation outcomes. RESULTS: Nine patients underwent transplantation (median age, 38 months; 7 hepatoblastoma, 2 undifferentiated mesenchymal sarcoma). All were assessed unresectable at presentation. After chemotherapy, 7 remained unresectable and had primary transplantation, 1 developed chemotherapy-related liver failure, necessitating emergent transplantation, and 1 was deemed resectable, requiring rescue transplantation after local recurrence. Using a timely living/cadaver donor graft acquisition strategy relative to chemotherapy, median waiting time from listing was 8 days. After transplantation, 3 of 9 had chemotherapy, with side effects dictating discontinuation in 2; 6 of 9 had no chemotherapy, with 2 developing distant metastases, 1 of whom died 12 months posttransplantation. Median follow-up was 3.08 years. Overall survival was 89%. CONCLUSIONS: Primary transplantation can be highly successful in children with hepatic tumors. These outcomes compare favorably with multicenter studies, where waiting-list deaths are reported and survival after rescue transplantation is poor. We encourage timely transplantation in the setting of questionably resectable tumors or evidence of chemotherapy resistance. The necessity of posttransplantation chemotherapy is questioned. Consultation with a transplantation program before chemotherapy should avoid inappropriate attempts at resection and allow appropriate planning of transplantation in relation to chemotherapy.