The purpose of this study was to evaluate the efficiency of treatment of living-related liver transplantation (LRLT) with the parental heterozygote carrier graft in children with Wilson disease.

We retrospectively evaluated 7 children with Wilson disease who had received liver transplantation from 1994 to 2002 at Asan Medical Center. All the donors were parental. Liver functions, Kayser-Fleischer ring, and other factors regarding to copper metabolism were analyzed.

Of the 7 children, 5 had fulminant hepatitis and 2 had decompensated liver cirrhosis irresponsive to medical therapy. All donors being parental, all grafts came to be heterozygote carrier grafts. Survival rate was 100% in those 7 children, 87% in all children with liver transplantation in the same period, and 84% in children with non-metabolic liver disease. After liver transplantation, all 7 children could stop low copper diet and penicillamine therapy and their AST, total bilirubin and prothrombin time were recovered to normal. After liver transplantation, ceruloplasmin and serum copper levels were also recovered to normal. A marked reduction in 24 hr-urinary copper excretion was observed in all recipients after transplantation. During follow-up, Kayser-Fleischer rings resolved completely after LRLT in 5 children and partially in 1 child.

We concluded that living-related liver tranplantation in children with Wilson disease with parental heterozygote carrier graft is an effective treatment modality.