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Current Gastroenterology Reports

, Volume 8, Issue 3, pp 215–223 | Cite as

Liver transplantation for non-hepatotoxic inborn errors of metabolism

  • William R. Treem
Article

Abstract

Hepatic-based inborn errors of metabolism are targets for treatment with liver transplantation in children, in whom the metabolic defect causes irreversible damage to the liver. However, certain metabolic defects originate with enzyme deficiencies localized in the liver but then give rise to toxic intermediates that damage extrahepatic organs without any significant compromise of general liver function. Here, the rationale of using liver transplantation to replace an organ that is functioning normally except for a specific metabolic pathway raises difficult questions about indications for transplantation, timing, amount of replacement tissue needed to correct the defect, and whether heterozygote parents are suitable living donors for liver transplantation in their affected children. This review explores these questions and others, including the role of hepatocyte transplantation, in this select group of disorders. Until the promise of specific gene or enzyme replacement therapy is realized, liver and hepatocyte transplantation offers the best chance of achieving metabolic control in these challenging patients.

Keywords

Liver Transplantation Glycogen Storage Disease Glycogen Storage Disease Type Primary Hyperoxaluria Urea Cycle Disorder 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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Copyright information

© Current Science Inc 2006

Authors and Affiliations

  1. 1.Division of Pediatric Gastroenterology, Hepatology, and NutritionSUNY Downstate Medical CenterBrooklynUSA

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