Initial liver transplantation for unresectable hepatoblastoma after chemotherapy.

BACKGROUND: SIOPEL protocols have recommended liver transplantation for unresectable hepatoblastoma (HBL) after chemotherapy in absence of visible extrahepatic disease. METHODS: This retrospective single center study includes 13 children treated following SIOPEL 3 or 4 protocols who underwent orthotopic liver transplantation (OLT) for HBL between February 2001 and May 2009. RESULTS: Twelve patients had PRETEXT IV HBL, one had PRETEXT II P + HBL, two had pulmonary metastasis at diagnosis. Extra hepatic vascular involvement was present in seven patients (two vena cava, four main portal vein). Twelve patients received a deceased donor organ graft; wait time to OLT was 16 days (1-50 days). One patient received a living donor graft. Four patients did not undergo post-OLT chemotherapy because of major post-OLT surgical complications. Mean follow up was 3.1 years (1-5 years). Ten patients are alive, eight in first complete remission (CR), one is in second CR after two surgical pulmonary metastasis were removed, the latter is in second CR after a surgery for excision of two local recurrences and re-OLT for a secondary HBL in the first graft. Three patients died (two from tumor recurrence, one from cardiac failure after second OLT). Overall survival at 1 and 4 years was 100% and 83.3%. CONCLUSIONS: Application of SIOPEL protocols for treatment of HBL in a specialized multidisciplinary team with access to liver transplantation has resulted in excellent survival. Initial extrahepatic disease should not be considered a contraindication. Future refinements of the protocol need to be considered to reduce toxicity.