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标题：Use of waxy maize heat modified starch in the treatment of children between 2 and 5 years with glycogen storage disease type I: A retrospective study
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作者：Ghada Hijazi ; Nisha Pai ; Laura L. Nagy 等
期刊名称：Molecular Genetics and Metabolism Reports
印刷版ISSN：2214-4269
出版年度：2019
卷号：21
页码：1-6
DOI：10.1016/j.ymgmr.2019.100536
出版社：Elsevier B.V.
摘要：Background Glycogen storage disease type I (GSDI) is caused by deficiency of the enzyme glucose-6-phosphatase or glucose-6-phosphate transporter. Mainstay of treatment is provision of uncooked cornstarch (and/or continuous nocturnal pump feed (CNPF) to maintain normoglycemia). Waxy maize heat modified starch (WMHMS) is another treatment option to maintain normoglycemia overnight. Our objective was to describe our experience treating children 2–5 years of age with GSDI using WMHMS overnight. Method This is a retrospective case series review ( n  = 5) comparing the overnight feeding regimen and biochemical control one year before and after nocturnal WMHMS therapy. The WMHMS trial, in which blood glucose and lactate levels were monitored hourly, is reported in detail. Results Most patients successfully transitioned to nocturnal WMHMS feeds. These patients had stable glucose and lactate throughout the overnight period, permitting a fasting period of 6.5–8 h overnight. Within the time period studied, WMHMS appeared to have improved overnight control of blood glucose levels with fewer reported episodes of hypoglycemia compared to CNPF. Conclusion WMHMS can be an effective substitute treatment to achieve stable nocturnal glucose control in children younger than five years of age. A larger multicenter prospective study is recommended to establish stronger evidence of the efficacy and safety of using WMHMS in treatment of young children with GSDI.
关键词：Glycogen storage disease (GSD) ; Uncooked cornstarch (UCCS) ; Waxy maize heat modified starch (WMHMS) ; Continuous nocturnal pump feed (CNPF). ; GSD Glycogen storage disease ; UCCS uncooked cornstarch ; WMHMS waxy maize heat modified starch ; CNPF continuous nocturnal pump feed ; TGs triglycerides ; abdominal US abdominal ultrasound ; ALT alanine aminotransferase ; AST aspartate aminotransferase ; kg kilogram ; cm centimeter ; BMI body mass index ; GI gastrointestinal ; hrs hours