The potential of dietary treatment in patients with glycogen storage disease type IV. Issue 3 (21st December 2020)

• Record Type: Journal Article
• Authors: Derks, Terry G. J.; Peeks, Fabian; de Boer, Foekje; Fokkert‐Wilts, Marieke; van der Doef, Hubert P. J.; van den Heuvel, Marius C.; Szymańska, Edyta; Rokicki, Dariusz; Ryan, Patrick T.; Weinstein, David A.
• Is Part Of: Journal of inherited metabolic disease. Volume 44:Issue 3(2021)
• Page Start: 693
• Page End: 704
• Publication Date: 2020-12-21
• Subjects: dietary intervention -- glycogen storage disease -- glycogen storage disease type IV -- inherited metabolic disease -- liver transplantation; Metabolism, Inborn errors of -- Periodicals; Metabolism -- Disorders -- Periodicals; 616.39042
• DOI: 10.1002/jimd.12339
• Languages: English
• ISSNs: 0141-8955

Abstract

Abstract: 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. Abstract :