Long‐term outcome after liver transplantation in children with type 1 glycogen storage disease

Patients with GSD type 1 (von Gierke disease) are initially managed medically to maintain normoglycemia. However, if they do not achieve good metabolic control, LT is then considered. We describe the long‐term outcome of 6 children with GSD type 1 who underwent LT. Retrospective chart review of the...

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Bibliographic Details
Published in:Pediatric transplantation 2021-03, Vol.25 (2), p.e13872-n/a
Main Authors: Yuen, Wing Yan, Quak, Seng Hock, Aw, Marion M., Karthik, Sivaramakrishnan Venkatesh
Format: Article
Language:English
Subjects:
Adolescent
Allopurinol
Child
Child, Preschool
Children
Enzymes
Female
Follow-Up Studies
Glycogen
glycogen storage disease
Glycogen Storage Disease Type I - surgery
Humans
Hypercholesterolemia
Hyperlactatemia
Hypertriglyceridemia
Hyperuricemia
Hypoglycemia
Infant
Liver Transplantation
Liver transplants
Male
metabolic control
Metabolism
paediatrics
Retrospective Studies
Storage diseases
Transplants & implants
Treatment Outcome
von Gierke disease
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Summary:Patients with GSD type 1 (von Gierke disease) are initially managed medically to maintain normoglycemia. However, if they do not achieve good metabolic control, LT is then considered. We describe the long‐term outcome of 6 children with GSD type 1 who underwent LT. Retrospective chart review of the data of 6 children with GSD type 1 who underwent LT at National University Hospital, Singapore, from May 1998 to October 2018, was performed. The median (IQR) age at diagnosis of the GSD was 1 year (0.92‐5.50) and at transplant was 13.88 years (11.46‐16.38). All of the patients had elevated liver enzymes, hypercholesterolemia, hypertriglyceridemia, and hyperlactatemia prior to transplant. All of the patients are alive at the time of analysis and follow‐up. None of them required a re‐transplant. For the three patients who had hypoglycemia pretransplant, there was no recurrence post‐transplant. All of the patients had normalization of liver enzymes by 1 year post‐transplant. Long‐term outcome of patients with GSD who underwent LT has been positive with improvement in metabolic control for most patients. We report the unusual finding of two siblings with persistent hyperuricemia post‐transplant requiring allopurinol.
ISSN:1397-3142
1399-3046
DOI:10.1111/petr.13872