Outcomes of Liver Transplantation for Unresectable Liver Malignancy in Children.

Abstract

Background: Unresectable liver cancer accounts for an increasing number of liver transplants in children. Purpose/Methods: A retrospective, single-center outcomes assessment of 76 children <21 years who received liver transplantation for unresectable liver malignancy over a 32-year period between 1981-2013. Results: Malignancies included hepatoblastoma (HBL)-43, hepatocellular cancer (HCC)-26, other primary-4 (embryonal sarcoma-2, rhabdoid-1, angiosarcoma-1), and metastastic tumors (mets)-3 (neuroendocrine-2, pseudopapillary pancreatic tumor-1). Children with hepatoblastoma and other primary tumors were younger compared with HCC and mets (median age 2.7 vs 4.4 vs 9.6 vs 16.4 years, respectively, p=0.01). Greater use of technical variant grafts (34 vs 11%, p=0.049) and living donors (20 vs 4%, p=0.079, NS) occurred in HBL compared with HCC. Pre-existing liver disease was less common in HBL (2/44, 5%, biliary atresia-1, cryoptogenic cirrhosis-1) than HCC (18/26, 69%, p=0.0001). In HCC, these diseases included tyrosinemia-5, familial cholestasis-3, bile duct disorders-4 (biliary atresia-1, choledochal cyst-1, neonatal cholestasis-1, bile duct paucity-1) hepatitis B-2, metabolic/cryptogenic cirrhosis-4. Recurrence-free survival was better among HBL/HCC with pre-existing liver disease (18/20, 90%, vs 39/50, 78%, p=0.33, NS) and among HCC first found in explants (14/15, 93.3% vs 4/11, 36.6%, p=0.003) compared with those children in whom HBL or HCC was the primary diagnosis. Graft thrombosis required retransplantation in two children. The distribution of causes of death (recurrence: infection/PTLD: technical: unknown) was 5: 1: 1: 1 in HBL, and 8: 4: 1: 4 in HCC. In other groups, one death occurred due to recurrent angiosarcoma. At median follow-up of 5.7 years (range 8 days to 26 years) actual recurrence-free and overall patient survival was 88.6% and 78% in HBL, 69% and 34.6% in HCC, 75% and 75% in other primary, and 100% and 100% in mets. Conclusions: Unresectable pediatric liver malignancy is effectively treated with liver transplantation. Concomittant HCC/HBL in congenital cholestatic/cirrhotic end-stage liver disease argues for improved surveillance and timely transplantation to optimize survival.