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为患有基因缺陷的心脏提供额外能量:ENERGY试验。

Extra energy for hearts with a genetic defect: ENERGY trial.

作者信息

van Driel B O, van Rossum A C, Michels M, Huurman R, van der Velden J

机构信息

Department of Physiology, Amsterdam Cardiovascular Sciences, Amsterdam UMC, location VUmc, Amsterdam, The Netherlands.

Department of Cardiology, Amsterdam Cardiovascular Sciences, Amsterdam UMC, location VUmc, Amsterdam, The Netherlands.

出版信息

Neth Heart J. 2019 Apr;27(4):200-205. doi: 10.1007/s12471-019-1239-0.

Abstract

AIMS

Previous studies have shown that hypertrophic cardiomyopathy mutation carriers have a decreased myocardial energy efficiency, which is thought to play a key role in the pathomechanism of hypertrophic cardiomyopathy (HCM). The ENERGY trial aims to determine whether metabolic drugs correct decreased myocardial energy efficiency in HCM mutation carriers at an early disease stage.

METHODS

40 genotype-positive, phenotype-negative MYH7 mutation carriers will be treated for two months with trimetazidine or placebo in a double-blind randomised study design. Directly before and after treatment, study subjects will undergo an [C]-acetate positron emission tomography/computed tomography (PET/CT) and cardiac magnetic resonance (CMR) scan to measure myocardial energy efficiency. Myocardial efficiency will be calculated as the amount of oxygen the heart consumes to perform work.

CONCLUSION

The ENERGY trial will be the first proof of concept study to determine whether metabolic drugs are a potential preventive therapy for HCM. Given that trimetazidine is already being used in clinical practice, there is large potential to swiftly implement this drug in HCM therapy.

摘要

目的

既往研究表明,肥厚型心肌病突变携带者心肌能量效率降低,这被认为在肥厚型心肌病(HCM)的发病机制中起关键作用。ENERGY试验旨在确定代谢药物能否在疾病早期纠正HCM突变携带者降低的心肌能量效率。

方法

在一项双盲随机研究设计中,40名基因型阳性、表型阴性的MYH7突变携带者将接受曲美他嗪或安慰剂治疗两个月。在治疗前后,研究对象将接受[C] - 醋酸盐正电子发射断层扫描/计算机断层扫描(PET/CT)和心脏磁共振(CMR)扫描,以测量心肌能量效率。心肌效率将以心脏做功所消耗的氧量来计算。

结论

ENERGY试验将是第一项确定代谢药物是否为HCM潜在预防性治疗的概念验证研究。鉴于曲美他嗪已在临床实践中使用,在HCM治疗中迅速应用这种药物具有很大潜力。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/276e/6439046/083e9b34ee87/12471_2019_1239_Fig1_HTML.jpg

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