Liver transplantation in metabolic disorders

Liver transplantation in pediatric patients represents about 10% of a total of 23,000 transplantations registered in the European Liver Transplantatio n Register (ELTR) since 1968. The pediatric patients show a specific spectr um of indications with cholestatic liver disorders ranking first, followed by hepatic based metabolic disorders. There has been a significant improvem ent of survival in transplantation since the early 80ies. The overall survi val standard is nowadays in the range of 80%. There is a trend towards even better results in metabolic disorders. The clinical presentation of liver disease caused by metabolic disorders sh ows a wide range from acute liver, cerebral, cardiac and renal failure to c hronic end stage liver, kidney and heart disease potentially complicated by hepatocellular carcinoma. In may cases, the diagnosis of a underlying meta bolic disorder is very difficult and time consuming so the decision to do a liver transplantation may be necessary before a final diagnosis is establi shed. Having these problems in mind, the consideration of absolute and relative c ontraindications for liver transplantation in metabolic disorders is even m ore difficult than it is already in cholestatic or inflammatory liver disor ders. the individual evaluation of a patient suffering from a hepatic metab olic must consider in addition the often dramatic restriction of quality of life sue to rigorous dietary restrictions or other therapies. This makes c lear that suitable methods to measure quality of life must be developed and applied in order to fulfill this goal. The extension of indications for liver transplantation even to disorders wi th only partial defects in otherwise healthy livers was possible by using i nnovative surgical techniques such as partial, living related, split, in si tu split and auxiliary orthotopic transplantation. These techniques allowed to reduce the mortality on pediatric waiting lists significantly without r estricting the general donor pool. however, living related liver transplant ation is handicaped by the heterozygous status of the parent donor. This pl ays a role especially in patients with progressive familial intraheptic cho lestasis (PFIC) and Wilson's disease.