Liver transplantation for pediatric metabolic disease

Liver transplantation (LTx) was initially developed as a therapy for liver diseases known to be associated with a high risk of near-term mortality but is based upon a different set of paradigms for inborn metabolic diseases. As overall outcomes for the procedure have improved, LTx has evolved into an attractive approach for a growing number of metabolic diseases in a variety of clinical situations. No longer simply life-saving, the procedure can lead to a better quality of life even if not all symptoms of the primary disorder are eliminated. Juggling the risk-benefit ratio thus has become more complicated as the list of potential disorders amenable to treatment with LTx has increased. This review summarizes presentations from a recent conference on metabolic liver transplantation held at the Children's Hospital of Pittsburgh of UPMC on the role of liver or hepatocyte transplantation in the treatment of metabolic liver disease. •Overall outcomes for metabolic liver transplant have improved in the past 3 decades.•LTx can lead to a better quality of life even if not all symptoms of the primary disorder are eliminated.•Risk-benefit ratio of LTx must be carefully evaluated.•Hepatocyte transplantation may be a viable alternative to whole organ replacement in some disorders.