Liver transplantation for Wilson's disease: A single-center experience

Wilson's disease is a hereditary defect in copper excretion leading to the accumulation of copper in the tissues, with subsequent tissue damage. The m ost serious sequela is that of progressive central nervous system involveme nt. The use of orthotopic liver transplantation (OLT) has been controversia l for those patients with neurological symptoms attributed to Wilson's dise ase. The aim of this study is to determine the effectiveness of OLT for pat ients with Wilson's disease, including those with neurological involvement attributed to copper accumulation in the central nervous system. OLT was pe rformed in 45 patients (19 men [42.2%], 26 women [57.8%]) with Wilson's dis ease between 1971 and 1993 who were followed up for at least 4 years. The a ge at diagnosis of Wilson's disease ranged from 3 to 41 years (mean, 17.7 /- 7.4 years). The age at OLT ranged from 8 to 52 years (mean, 22.3 +/- 9.4 years). Nineteen patients (42.2%) were aged younger than 18 years at OLT, The indications for OLT included chronic hepatic failure in 15 patients (33 .3%) and fulminant (FHF) or subfulminant hepatic failure in 30 patients (66 .6%), All but 1 of the 19 pediatric patients (94.7%) were in the latter gro up. Twenty-five patients (55.5%) were receiving D-penicillamine, 9 patients for more than 1 year; none of the patients treated long term presented as FHF. Thirty-three patients (73.3%) survived more than 5 years after OLT. Fo urteen patients (31%) died during the posttransplantation period; 7 of the 14 patients (50%) were aged younger than 18 years. Twelve patients died dur ing the first 3 months after OLT of complications of disease and surgery, 1 0 of whom underwent transplantation for FHF, The other 2 patients died 6 an d 9 years after transplantation of infectious problems. Eleven patients (24 .4%) required retransplantation because of a primary nonfunctioning graft ( n = 6), chronic rejection (n = 4), and hepatic artery thrombosis (n = 1). S eventeen patients (37.7%) presented with neurological abnormalities; 14 pat ients with Wilsonian neurological manifestations and 3 patients with compon ents of increased intracranial pressure. Ten of the 13 surviving patients w ith hepatic insufficiency and neurological abnormalities at OLT showed sign ificant neurological improvement. Our experience shows OLT is a lifesaving procedure in patients with end-stage Wilson's disease and is associated wit h excellent long-term survival. The neurological manifestation of the disea se can improve significantly after OLT. Earlier transplantation in patients with an unsatisfactory response to medical treatment may prevent irreversi ble neurological deterioration and less satisfactory improvement after OLT. Copyright (C) 1999 by the American Association for the Study of Liver Dise ases.