The potential of dietary treatment in patients with glycogen storage disease type IV,Journal of Inherited Metabolic Disease

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The potential of dietary treatment in patients with glycogen storage disease type IV
Journal of Inherited Metabolic Disease ( IF 4.2 ) Pub Date : 2020-12-17 , DOI: 10.1002/jimd.12339
Terry G J Derks ₁ , Fabian Peeks ₁ , Foekje de Boer ₁ , Marieke Fokkert-Wilts ₁ , Hubert P J van der Doef ₂ , Marius C van den Heuvel ₃ , Edyta Szymańska ₄ , Dariusz Rokicki ₅ , Patrick T Ryan ₆ , David A Weinstein _{6,

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Affiliation

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.

中文翻译：

IV型糖原贮积病患者饮食治疗的潜力

关于糖原贮积病 (GSD) IV 型饮食治疗的文献很少，也没有正式的指南。传统上，肝移植被认为是 GSD IV 的唯一治疗选择。鉴于饮食治疗其他肝脏形式的 GSD 的成功，我们启动了这项观察性研究，以评估限制糖原积累的医学饮食的结果。提供了来自三个中心的 15 名 GSD IV 患者的临床、饮食、实验室和成像数据。医学饮食可能有可能延迟或预防肝移植、促进生长并使血清转氨酶正常化。个人护理计划旨在避免高血糖、低血糖和/或高酮症，以分别最大程度地减少糖原积累和分解代谢。

更新日期：2020-12-17

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