Bhattacharya Kaustuv, Mundy Helen, Lilburn Maggie F, Champion Michael P, Morley David W, Maillot François
Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, WC1N 3BG, London, UK.
Discipline of Paediatrics and Child Health, The Children's Hospital at Westmead Clinical School, University of Sydney, Sydney, Australia.
Orphanet J Rare Dis. 2015 Feb 15;10:18. doi: 10.1186/s13023-015-0229-6.
Uncooked corn-starch (UCCS) has been the mainstay of therapy for the hepatic glycogen storage diseases (GSD) but is not always effective. A new starch (WMHMS) has demonstrated a more favourable short-term metabolic profile.
To determine efficacy and safety of a new uncooked starch (WMHMS) compared to UCCS over 16 weeks treatment with each.
A double-blind cross-over study of 10 adults (aged 16 - 38 years, six male) with GSD Ia and Ib. After an individualised fast, subjects were randomised to take a 50 g starch-load of either WMHMS or UCCS. Starch-loads terminated when blood glucose was < 3.0 mmol/L or the subject felt subjectively hypoglycaemic. Anonymous biochemical profiles were assessed by 2 investigators and a starch administration schedule recommended. Each starch was delivered in coded sachets and intake was monitored for the following 16 weeks. After a washout period, the protocol was repeated with the alternative product.
4 subjects failed to establish therapy on the cross-over limb. Data from 7 paired starch load showed: longer median fasting duration with WMHMS (7.5 versus 5 hours; p = 0.023), slower decrease in the glucose curve (0.357 versus 0.632 mmol/hr p = 0.028) and less area under insulin curves for the first 4 hours (p = 0.03). Two of six subjects took 50% or less WMHMS compared to UCCS and one took more. Plasma triglycerides, cholesterol and uric acid were unchanged after each study phase.
WMHMS leads to significant reduction in insulin release and reduced starch use in some GSD patients.
生玉米淀粉（UCCS）一直是肝糖原贮积病（GSD）治疗的主要手段，但并非总是有效。一种新的淀粉（WMHMS）已显示出更有利的短期代谢特征。
确定一种新的生淀粉（WMHMS）与UCCS相比，在16周治疗期内的疗效和安全性。
对10名患有GSD Ia和Ib的成年人（年龄16 - 38岁，6名男性）进行双盲交叉研究。在个体化禁食后，受试者被随机分配服用50克WMHMS或UCCS的淀粉负荷量。当血糖<3.0 mmol/L或受试者主观感觉低血糖时，停止淀粉负荷。由2名研究人员评估匿名生化指标并推荐淀粉给药方案。每种淀粉均装在编码小袋中提供，并在接下来的16周内监测摄入量。经过洗脱期后，用另一种产品重复该方案。
4名受试者在交叉阶段未能建立治疗。来自7对淀粉负荷的数据显示：WMHMS的中位禁食持续时间更长（7.5小时对5小时；p = 0.023），葡萄糖曲线下降更慢（0.357对0.632 mmol/小时，p = 0.028），前4小时胰岛素曲线下面积更小（p = 0.03）。与UCCS相比，6名受试者中有2名服用的WMHMS为50%或更少，1名服用更多。每个研究阶段后血浆甘油三酯、胆固醇和尿酸均无变化。
WMHMS可使一些GSD患者的胰岛素释放显著减少，淀粉用量降低。
