Liver transplantation for pediatric metabolic disease

Journal Article

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. © 2014.

Full Text

Duke Authors

Cited Authors

  • Mazariegos, G; Shneider, B; Burton, B; Fox, IJ; Hadzic, N; Kishnani, P; Morton, DH; Mcintire, S; Sokol, RJ; Summar, M; White, D; Chavanon, V; Vockley, J

Published Date

  • 2014

Published In

Volume / Issue

  • 111 / 4

Start / End Page

  • 418 - 427

International Standard Serial Number (ISSN)

  • 1096-7192

Digital Object Identifier (DOI)

  • 10.1016/j.ymgme.2014.01.006