Glycogen Storage Disease Program, Connecticut Childrens Medical Center, Hartford, USA.
School of Medicine, Department of Pediatrics, University of Connecticut, Farmington, CT, USA.
Orphanet J Rare Dis. 2024 Jul 9;19(1):258. doi: 10.1186/s13023-024-03274-y.
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.
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.
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.
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.
低血糖是所有肝糖原贮积病（GSD）的主要表现。2008 年，报道了一种缓释蜡质玉米淀粉 Glycosade，可替代生玉米淀粉（UCCS），延长 GSD 人群的禁食时间。迄今为止，（a）幼儿、（b）酮症型 GSD 和（c）日间用药的经验很少。Glyde 研究是一项前瞻性、全球性的计划，旨在测试 Glycosade 在更广泛和更多样化的人群中的疗效和耐受性。
一项随机、双盲交叉禁食研究评估了 Glycosade 与玉米淀粉相比的耐受性和疗效，涵盖了不同疾病类型和年龄。参与者和临床医生选择他们认为更好的产品，同时仍然保持盲法。对参与者进行了 2 年的随访，以评估长期代谢控制、生长和生活质量。
共有 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 GSD I；30 名 GSD III、VI、IX）。Glycosade 改善了 GSD I 的禁食时间和酮症形式的无酮禁食时间。69%的参与者选择了慢性 Glycosade 治疗。在 2 年的慢性治疗阶段，使用 Glycosade 治疗的患者使用的治疗剂量更少，而代谢控制的标志物保持稳定。
Glyde 研究是第一项多中心国际试验，证明了 Glycosade 在一个多样化的国际人群中对大量肝糖原贮积病患者的疗效和耐受性。每天使用更少的治疗剂量并避免夜间治疗可能会提高依从性、安全性和生活质量，而不会牺牲代谢控制。
