LIVER TRANSPLANTATION FOR HEMOCHROMATOSIS, WILSON'S DISEASE, AND OTHER METABOLIC DISORDERS

Abstract

Liver transplantation provides an effective means for replacing a failing liver, in addition to correcting the underlying abnormality in many metabolic disorders. Results of liver transplantation for metabolic diseases have been generally encouraging, with the exception of hereditary hemochromatosis, in which infectious and cardiac complications appear to increase post-transplant mortality. Better pretransplant diagnosis of hemochromatosis, utilizing the recently identified putative gene, may help reduce post-transplant complications. In metabolic diseases, improved understanding of the underlying genetic and molecular defects will lead to advances in medical therapy and perhaps a decreased need for liver transplantation. NTBC therapy for hereditary tyrosinemia and purified glucocerebroside therapy for Gaucher disease are two such examples. The prospects of gene therapy are being actively pursued for many metabolic diseases, such as CF, hemophilia, and familial hypercholesterolemia. Until such investigation leads directly to clinical practice, however, liver transplantation remains an effective option for therapy for a wide range of metabolic diseases.