Long-term personalized high-protein, high-fat diet in pediatric patients with glycogen storage disease type IIIa: Evaluation of myopathy, metabolic control, physical activity, growth, and dietary compliance

IF 4.2 2区 医学 Q1 ENDOCRINOLOGY & METABOLISM
Sema Kalkan Uçar, Yasemin Atik Altınok, Yelda Mansuroglu, Ebru Canda, Havva Yazıcı, Merve Yoldaş Çelik, Fehime Erdem, Ayşe Yüksel Yanbolu, Zülal Ülger, Mahmut Çoker
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Abstract

Dietary lipid manipulation has recently been proposed for managing glycogen storage disease (GSD) type IIIa. This study aimed to evaluate the myopathic, cardiac, and metabolic status, physical activity, growth, and dietary compliance of a personalized diet high in protein and fat for 24 months. Of 31 patients with type IIIa GSD, 12 met the inclusion criteria. Of these, 10 patients (mean age 11.2 ± 7.4 years) completed the study. Patients were prescribed a personalized high-protein, high-fat diet, comprising 3.0–3.5 g/kg/day of protein and 3.0–4.5 g/kg/day of fat, constituting 18.5%–28% and 70.5%–75.7% of daily energy, respectively. Dietary compliance was ensured and assessed via the regular administration of questionnaires. Our results revealed consistent and significant decreases of 22%, 54%, and 30% in the creatinine kinase, creatine kinase–myocardial band, and lactate dehydrogenase levels, respectively. Echocardiography revealed improvements in the Z-scores of the left ventricular mass and interventricular septum thickness. A significant increase in body muscle mass was observed, and a higher score was achieved using the Daily Activity Questionnaire. Growth monitoring revealed an arrest in the height-SDS at the 6th and 12th months, followed by subsequent improvement at the end of the second year. A gradual and persistent decline in the periods of hypo- and hyperglycemia has been reported. Biotinidase activity decreased, whereas hepatosteatosis increased and then decreased by the end of the study. Implementing a high-protein, high-fat diet and monitoring key parameters in patients with type IIIa GSD can lead to myopathic and cardiac improvements and increased physical activity.

Abstract Image

糖原贮积症 IIIa 型儿科患者的长期个性化高蛋白、高脂肪饮食:评估肌病、代谢控制、体力活动、生长和饮食依从性
最近,有人提出通过饮食调脂来控制糖原贮积病(GSD)IIIa 型。本研究旨在评估高蛋白和高脂肪个性化饮食 24 个月的肌病、心脏和代谢状况、体力活动、生长和饮食依从性。在 31 名 IIIa 型 GSD 患者中,有 12 人符合纳入标准。其中,10 名患者(平均年龄为 11.2±7.4 岁)完成了研究。研究人员为患者制定了个性化的高蛋白、高脂肪饮食,其中蛋白质含量为 3.0-3.5 克/千克/天,脂肪含量为 3.0-4.5 克/千克/天,分别占每日能量的 18.5%-28% 和 70.5%-75.7% 。通过定期发放调查问卷来确保和评估饮食依从性。我们的研究结果显示,肌酸激酶、肌酸激酶-心肌带和乳酸脱氢酶水平分别持续显著下降了 22%、54% 和 30%。超声心动图显示,左心室质量和室间隔厚度的 Z 值均有所改善。身体肌肉质量明显增加,日常活动问卷得分也有所提高。生长监测显示,身高-SDS 在第 6 个月和第 12 个月出现停滞,随后在第二年年底有所改善。据报道,低血糖和高血糖的持续时间逐渐缩短。研究结束时,生物素酶活性下降,而肝脂肪变性增加,随后又下降。对 IIIa 型 GSD 患者实施高蛋白、高脂肪饮食并监测关键参数,可改善肌病和心脏状况,增加体力活动。
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来源期刊
Journal of Inherited Metabolic Disease
Journal of Inherited Metabolic Disease 医学-内分泌学与代谢
CiteScore
9.50
自引率
7.10%
发文量
117
审稿时长
4-8 weeks
期刊介绍: The Journal of Inherited Metabolic Disease (JIMD) is the official journal of the Society for the Study of Inborn Errors of Metabolism (SSIEM). By enhancing communication between workers in the field throughout the world, the JIMD aims to improve the management and understanding of inherited metabolic disorders. It publishes results of original research and new or important observations pertaining to any aspect of inherited metabolic disease in humans and higher animals. This includes clinical (medical, dental and veterinary), biochemical, genetic (including cytogenetic, molecular and population genetic), experimental (including cell biological), methodological, theoretical, epidemiological, ethical and counselling aspects. The JIMD also reviews important new developments or controversial issues relating to metabolic disorders and publishes reviews and short reports arising from the Society''s annual symposia. A distinction is made between peer-reviewed scientific material that is selected because of its significance for other professionals in the field and non-peer- reviewed material that aims to be important, controversial, interesting or entertaining (“Extras”).
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