Copenhagen Neuromuscular Center, Department of Neurology, Copenhagen University Hospital, Rigshospitalet, DK-2100 Copenhagen, Denmark.
Department of Clinical Medicine, University of Copenhagen, DK-2200 Copenhagen, Denmark.
Nutrients. 2023 Feb 7;15(4):843. doi: 10.3390/nu15040843.
The low-carbohydrate ketogenic diet (LCKD) has attracted increased attention in recent years as a potential treatment option for individuals with McArdle disease (glycogen storage disease type V), and despite the absence of strong scientific evidence of the LCKD's benefits, increased numbers of individuals with McArdle disease have tried a LCKD. The objective of this study was to collect patient-reported experiences with a LCKD. We aimed to estimate the immediate prevalence of individuals that had tried a LCKD in an international McArdle disease cohort, and we aimed to report on the patient-reported experiences with the diet, both positive and negative. A total of 183 responses were collected from individuals with McArdle disease from 18 countries. We found that one-third of the cohort had tried a LCKD, and almost 90% experienced some degree of positive effect, with the most prominent effects on McArdle disease-related core symptoms (e.g., activity intolerance, muscle pain, and muscle fatigue). Adverse effects were rare and generally rated as mild to moderate. These patient-reported findings underline the need for randomized clinical trials to decisively determine if a LCKD is a suitable nutritional strategy for patients with McArdle disease. The results from this study can prompt and contribute to the design of such a clinical trial.
近年来，低碳水化合物生酮饮食（LCKD）作为麦卡德尔病（糖原贮积病 V 型）患者的潜在治疗选择引起了越来越多的关注，尽管缺乏 LCKD 益处的强有力科学证据，但越来越多的麦卡德尔病患者尝试了 LCKD。本研究的目的是收集患者对 LCKD 的体验报告。我们旨在估计在国际麦卡德尔病队列中尝试 LCKD 的个体的即时流行率，我们旨在报告饮食的患者报告体验，包括积极和消极的方面。从 18 个国家的麦卡德尔病患者中收集了 183 份回复。我们发现，三分之一的队列尝试了 LCKD，近 90%的患者体验到了某种程度的积极效果，对麦卡德尔病相关核心症状（如活动不耐受、肌肉疼痛和肌肉疲劳）的影响最为明显。不良反应罕见，通常为轻度至中度。这些患者报告的发现强调了需要进行随机临床试验来明确确定 LCKD 是否适合麦卡德尔病患者的营养策略。本研究的结果可以为这类临床试验的设计提供启示和帮助。
