The potential of dietary treatment in patients with glycogen storage disease type IV

There is paucity of literature on dietary treatment in glycogen storage disease (GSD) type IV and formal guidelines are not available. Traditionally, liver transplantation was considered the only treatment option for GSD IV. In light of the success of dietary treatment for the other hepatic forms of...

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Bibliographic Details
Published in:Journal of inherited metabolic disease 2021-05, Vol.44 (3), p.693-704
Main Authors: Derks, Terry G. J., Peeks, Fabian, Boer, Foekje, Fokkert‐Wilts, Marieke, Doef, Hubert P. J., Heuvel, Marius C., Szymańska, Edyta, Rokicki, Dariusz, Ryan, Patrick T., Weinstein, David A.
Format: Article
Language:English
Subjects:
Adolescent
Adult
Bile
Biomarkers
Child
Child, Preschool
Detoxification
Diet
dietary intervention
Dietary Supplements
Female
Glycogen
Glycogen - metabolism
glycogen storage disease
glycogen storage disease type IV
Glycogen Storage Disease Type IV - diet therapy
Glycogen Storage Disease Type IV - pathology
Homeostasis
Humans
Hyperglycemia
Hypoglycemia
Infant
inherited metabolic disease
Interdisciplinary Communication
Lactic acid
Liver
Liver - metabolism
Liver - pathology
Liver Transplantation
Liver transplants
Male
Original
Patients
Storage diseases
Treatment Outcome
Young Adult
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Summary:There is paucity of literature on dietary treatment in glycogen storage disease (GSD) type IV and formal guidelines are not available. Traditionally, liver transplantation was considered the only treatment option for GSD IV. In light of the success of dietary treatment for the other hepatic forms of GSD, we have initiated this observational study to assess the outcomes of medical diets, which limit the accumulation of glycogen. Clinical, dietary, laboratory, and imaging data for 15 GSD IV patients from three centres are presented. Medical diets may have the potential to delay or prevent liver transplantation, improve growth and normalize serum aminotransferases. Individual care plans aim to avoid both hyperglycaemia, hypoglycaemia and/or hyperketosis, to minimize glycogen accumulation and catabolism, respectively. Multidisciplinary monitoring includes balancing between traditional markers of metabolic control (ie, growth, liver size, serum aminotransferases, glucose homeostasis, lactate, and ketones), liver function (ie, synthesis, bile flow and detoxification of protein), and symptoms and signs of portal hypertension.
ISSN:0141-8955
1573-2665
DOI:10.1002/jimd.12339