Derks Terry G J, Overduin Ruben J, Grünert Sarah C, Rossi Alessandro
Department of Metabolic Diseases, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, the Netherlands.
UMCG Center of Expertise for Carbohydrate, Fatty Acid Oxidation and Ketone Bodies Disorders, University Medical Center Groningen, Groningen, the Netherlands.
J Inherit Metab Dis. 2025 May;48(3):e70040. doi: 10.1002/jimd.70040.
Continuous glucose monitoring (CGM) is increasingly used although not officially registered for the management of people living with liver glycogen storage diseases (GSDs). The aims of this study were twofold: (a) to investigate the current experiences of healthcare providers (HCPs), patients, and caregivers using CGM to monitor glucose concentrations in liver GSDs, and (b) to formulate consensus statements. Two web-based questionnaires were distributed, one for HCPs and one for patients and/or their caregivers. The questionnaires collected data on demographics and epidemiology, current use of CGM, and opinions and statements about CGM in GSDs. For the statements, respondents rated their agreement on a 5-point Likert scale, and the consensus level was set at 75%. One Hundred Fourteen HCPs (including 87 physicians and 26 dietitians) from 28 countries responded, representing care of approximately 3800 liver GSD patients. Additionally, 148 GSD patients and/or their caregivers from 21 countries responded, mainly representing GSD Ia (n = 50), GSD Ib (n = 56), GSD III (n = 14), and GSD IX (n = 18). The median age to consider starting to use CGM was 6 and 2 months for HCPs and GSD families, respectively. Out of 16 statements common to the two questionnaires, HCPs and patients/caregivers reached consensus on 12 statements in both groups. Use of CGM is considered standard of care by both HCPs and GSD families, but reimbursement of CGM devices is challenging. Compared to diabetes mellitus, CGM should be applied differently in liver GSDs. Consensus guidelines are warranted on the use of CGM in liver GSDs, both in routine healthcare and in clinical trials.
连续血糖监测(CGM)的使用越来越广泛,尽管它尚未正式注册用于肝糖原贮积病(GSD)患者的管理。本研究的目的有两个:(a)调查医疗服务提供者(HCP)、患者和护理人员目前使用CGM监测肝GSD患者血糖浓度的经验,(b)制定共识声明。我们分发了两份基于网络的问卷,一份给HCP,一份给患者和/或他们的护理人员。问卷收集了关于人口统计学和流行病学、CGM的当前使用情况以及关于GSD中CGM的意见和声明的数据。对于这些声明,受访者在5点李克特量表上对他们的同意程度进行评分,共识水平设定为75%。来自28个国家的114名HCP(包括87名医生和26名营养师)做出了回应,代表了大约3800名肝GSD患者的护理情况。此外,来自21个国家的148名GSD患者和/或他们的护理人员做出了回应,主要代表GSD Ia(n = 50)、GSD Ib(n = 56)、GSD III(n = 14)和GSD IX(n = 18)。HCP和GSD家庭考虑开始使用CGM的中位年龄分别为6岁和2个月。在两份问卷共有的16项声明中,HCP和患者/护理人员在两组的12项声明上达成了共识。HCP和GSD家庭都认为使用CGM是护理标准,但CGM设备的报销具有挑战性。与糖尿病相比,CGM在肝GSD中的应用应该有所不同。在常规医疗保健和临床试验中,都需要关于在肝GSD中使用CGM的共识指南。