Allogenic liver transplantation - a form of "gene therapy" in metabolic diseases. Munich results and review

Introduction: Liver transplantation is the method of choice for metabolic d iseases and end-stage liver failure. Methods: At the Klinikum Gro ss hadern we have performed liver transplantation for inborn errors of metabolism in 24 patients (5.3 % of all transplantations, 16 adults, age 39 +/- 13 years ; 8 children, age 9 +/- 3 years); 19 patients received a transplant for end -stage liver disease, and in 5 cases because of fulminant hepatic failure. Results: Twenty-four patients received 27 transplants. In 3 cases, a split- liver transplantation was performed; one patient received a combined lung-l iver graft. The 5-year survival rate for children is 100 % and for adults 6 8 %. Conclusions: Liver transplantation for inborn errors of metabolism not only replaces the diseased organ, but also leads to complete reversal of t he metabolic defect.