背景:低血糖是所有肝型糖原贮积病(GSD)的主要表现。2008年,Glycosade®,一种缓释蜡质玉米玉米淀粉,据报道,未煮熟的玉米淀粉(UCCS)的替代品可以延长GSD人群的禁食时间。迄今为止,在(一)幼儿中,发表的经验很少,(b)GSD的酮症形式,和(c)日间给药。Glyde研究是前瞻性的,在更广泛和更多样化的人群中测试Glycosade使用的功效和耐受性的全球倡议。
方法:进行了一项随机双盲交叉禁食研究,评估了不同疾病类型和年龄的糖与玉米淀粉相比的耐受性和疗效。参与者和临床医生选择了被认为优越的产品,虽然仍然失明。参与者随访2年,以评估长期代谢控制。增长,和生活质量。
结果:61名参与者(年龄2-62岁;59%为女性)入组,58名参与者完成了禁食研究(28名GSDI;30名GSDIII,VI,IX).糖血改善了GSDI中的禁食持续时间和酮症形式中没有酮症的禁食持续时间。69%的参与者选择了慢性糖表病的使用。那些在2年慢性期接受Glycosade治疗的患者使用了较少剂量的治疗,而代谢控制的标志物保持稳定。
结论:Glyde研究是第一个多中心国际试验,证明了Glycosade在不同国际人群的大量肝性GSD患者中的疗效和耐受性。每天使用更少的治疗剂量和避免过夜治疗的能力可以提高依从性,安全,在不牺牲代谢控制的情况下提高生活质量。
BACKGROUND: Hypoglycaemia is the primary manifestation of all the hepatic types of glycogen storage disease (GSD). In 2008, Glycosade®, an extended-release waxy maize cornstarch, was reported as an alternative to uncooked cornstarch (UCCS) which could prolong the duration of fasting in the GSD population. To date, there has been minimal published experience in (a) young children, (b) the ketotic forms of GSD, and (c) with daytime dosing. The Glyde study was created as a prospective, global initiative to test the efficacy and tolerance of Glycosade use across a broader and more diverse population.
METHODS: A randomised double-blind cross-over fasting study assessing the tolerance and efficacy of Glycosade compared with cornstarch was performed across disease types and ages. Participants and clinicians chose the product deemed superior, whilst still blinded. Participants were followed for 2 years to assess long-term metabolic control, growth, and quality of life.
RESULTS: Sixty-one participants (age 2-62 years; 59% female) were enrolled, and 58 participants completed the fasting studies (28 GSD I; 30 GSD III, VI, IX). Glycosade improved duration of fasting in GSD I and duration of fasting without ketosis in the ketotic forms. Chronic Glycosade use was chosen by 69% of participants. Those treated with Glycosade for the 2-year chronic phase used fewer doses of therapy while markers of metabolic control remained stable.
CONCLUSIONS: The Glyde study is the first multi-centre international trial demonstrating the efficacy and tolerance of Glycosade in a large cohort of hepatic GSD patients across a diverse international population. The ability to use fewer doses of therapy per day and avoidance of overnight therapy may improve compliance, safety, and quality of life without sacrificing metabolic control.