Introduction : liver and liver cell transplantation for inborn errors of liver metabolism

Inborn errors of metabolism affect around 1/900 life births. Most of these conditions are rare, but any physi- cian will face patients affected by one or another of these diseases. Management of patients with inborn errors of metab- olism is often complex and includes orphan medica- tions, very specific diets, special education. Beside life threatening conditions, a main concern is that long term intellectual prognosis of certain patients may be im- paired, especially for diseases such as urea cycle disor- ders, organic aciduria and amino acidopathies. Quality of life is often poor, due to anorexia, naso gastric feeding, poor variability of the diet and severe diet restriction, social eviction, special education re- quirements, frequent hospitalisations. Liver transplantation has now become a very success- ful procedure, and more than 90% of the children sur- vive on the long term. It remains however an heavy and radical procedure, and doctors and patients may be reluctant to undergo this operation for non vital indica- tions. In addition, new hopes and perspectives such as emerging cell therapy and prospects of gene therapy are now being considered in the decision making pro- cess (1;2) (3).