LIVER-TRANSPLANTATION FOR WILSONS-DISEASE

Background/Aims: As has been the case with other metabolic diseases of the liver in the last decade, orthotopic liver transplantation has be en applied to the treatment of Wilson's disease with increasing freque ncy. The experience at the University of Pittsburg with orthotopic liv er transplantation for Wilson's disease is reported. Methods: Between February 1981 and December 1991, 51 orthotopic liver transplants were performed on 39 patients (16 pediatric, 23 adults) with Wilson's disea se, Twenty-two patients were transplanted because of a presentation co -existent with fulminant hepatic failure, Seventeen presented with chr onic advanced liver disease with (n=9) or without (n=8) associated neu rologic dysfunction. Results: The rate of primary graft survival (n-39 ) was 73% and patient survival was 79.4%, No patient mortality occurre d beyond 3 weeks post-orthotopic liver transplantation. Survival was b etter for those with a chronic advanced liver disease presentation (90 %) than it was for those with a fulminant hepatic failure (73%) presen tation, but the difference was not statistically significant.Conclusio ns: 1) Currently, orthotopic liver transplantation is the treatment of choice for Wilson's disease presenting as fulminant hepatic hepatic f ailure; 2) orthotopic liver transplantation should be considered for p atients with Wilson's disease with advanced, chronic liver disease for whom no other therapy is possible; 3) orthotopic liver transplantatio n only partially corrects the underlying metabolic defect of patients with Wilson's disease and converts the copper kinetics from that chara cteristic of an individual affected with a homozygous disease to that of an individual who is an obligate heterozygote, thereby effecting a phenotypic cure.