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Liver transplantation in glycogen storage disease type I.

Abstract
Glycogen storage disease type I (GSDI), an inborn error of carbohydrate metabolism, is caused by defects in the glucose-6-transporter/glucose-6-phosphatase complex, which is essential in glucose homeostasis. Two types exist, GSDIa and GSDIb, each caused by different defects in the complex. GSDIa is characterized by fasting intolerance and subsequent metabolic derangements. In addition to these clinical manifestations, patients with GSDIb suffer from neutropenia with neutrophil dysfunction and inflammatory bowel disease.With the feasibility of novel cell-based therapies, including hepatocyte transplantations and liver stem cell transplantations, it is essential to consider long term outcomes of liver replacement therapy. We reviewed all GSDI patients with liver transplantation identified in literature and through personal communication with treating physicians. Our review shows that all 80 GSDI patients showed improved metabolic control and normal fasting tolerance after liver transplantation. Although some complications might be caused by disease progression, most complications seemed related to the liver transplantation procedure and subsequent immune suppression. These results highlight the potential of other therapeutic strategies, like cell-based therapies for liver replacement, which are expected to normalize liver function with a lower risk of complications of the procedure and immune suppression.
AuthorsSusanna J B Boers, Gepke Visser, Peter G P A Smit, Sabine A Fuchs
JournalOrphanet journal of rare diseases (Orphanet J Rare Dis) Vol. 9 Pg. 47 (Apr 09 2014) ISSN: 1750-1172 [Electronic] England
PMID24716823 (Publication Type: Journal Article, Review)
Topics
  • Adolescent
  • Child
  • Child, Preschool
  • Female
  • Glycogen Storage Disease Type I (surgery)
  • Humans
  • Liver Transplantation
  • Male
  • Young Adult

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