Liver transplantation for pediatric metabolic disease

Mazariegos, G.; Shneider, B.; Burton, B.; Fox, I. J.; Hadzic, N.; Kishnani, P.; Morton, D. H.; McIntire, S.; Sokol, R. J.; Summar, M.; White, D.; Chavanon, V.; Vockley, J.

Mol Genet Metab. 2014 Feb 6; 111(4):418-27


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.

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