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[脂质代谢的原发性疾病:它们在当前血脂异常指南中的地位及治疗创新]

[Primary disorders of lipid metabolism: their place in current dyslipidemia guidelines and treatment innovations].

作者信息

Klose G, Gouni-Berthold I, März W

机构信息

Praxis für Endokrinologie Dres. I. Van de Loo & K. Spieker, Gerold-Janssen-Str. 2A, 28359, Bremen, Deutschland.

Poliklinik für Endokrinologie, Diabetes und Präventivmedizin, Medizinische Fakultät und Uniklinik Köln, Universität zu Köln, Köln, Deutschland.

出版信息

Inn Med (Heidelb). 2023 Sep;64(9):895-906. doi: 10.1007/s00108-023-01524-y. Epub 2023 Jun 6.

Abstract

According to current guidelines, the selection and intensity of lipid-effective therapies are based on the risk to be treated. The sole clinical categories of primary and secondary prevention of cardiovascular diseases result in over- and under-treatment, which may be a contributory cause of incomplete implementation of current guidelines in everyday practice. For the extent of benefit in cardiovascular outcome studies with lipid-lowering drugs, the importance of dyslipdemia for the pathogenesis of atherosclerosis-related diseases is crucial. Primary lipid metabolism disorders are characterized by life-long increased exposure to atherogenic lipoproteins. This article describes the relevance of new data for low density lipoprotein-effective therapy: inhibition of proprotein convertase subtilisin/kexin type 9 (PCSK9), adenosine triphosphate (ATP) citrate lyase with bempedoic acid, and ANGPTL3 with special consideration of primary lipid metabolism disorders, which are insufficiently taken into account, or not taken into account at all, in current guidelines. This is due to their apparently low prevalence rate and thus the lack of large outcome studies. The authors also discuss the consequences of increased lipoprotein (a), which cannot be sufficiently reduced until the ongoing intervention studies examining antisense oligonucleotides and small interfering RNA (siRNA) against apolipoprotein (a) are completed. Another challenge in practice is the treatment of rare, massive hypertriglyceridemia, especially with the aim of preventing pancreatitis. For this purpose, the apolipoprotein C3 (ApoC3) antisense oligonucleotide volenasorsen is available, which binds to the mRNA for ApoC3 and lowers triglycerides by around three quarters.

摘要

根据当前指南,降脂治疗的选择和强度基于所要治疗的风险。心血管疾病一级和二级预防的单一临床分类导致治疗过度和治疗不足,这可能是当前指南在日常实践中未得到充分实施的一个促成因素。对于降脂药物在心血管结局研究中的获益程度而言,血脂异常在动脉粥样硬化相关疾病发病机制中的重要性至关重要。原发性脂质代谢紊乱的特征是终生暴露于致动脉粥样硬化脂蛋白的水平升高。本文描述了新数据对低密度脂蛋白有效治疗的相关性:抑制前蛋白转化酶枯草溶菌素/kexin 9型(PCSK9)、用贝派地酸抑制三磷酸腺苷(ATP)柠檬酸裂解酶以及抑制血管生成素样蛋白3(ANGPTL3),并特别考虑了原发性脂质代谢紊乱,而在当前指南中这些紊乱未得到充分考虑或根本未被考虑。这是由于它们的患病率明显较低,因此缺乏大型结局研究。作者还讨论了脂蛋白(a)升高的后果,在针对载脂蛋白(a)的反义寡核苷酸和小干扰RNA(siRNA)的正在进行的干预研究完成之前,脂蛋白(a)无法充分降低。实践中的另一个挑战是治疗罕见的重度高甘油三酯血症,尤其是以预防胰腺炎为目的。为此,可使用载脂蛋白C3(ApoC3)反义寡核苷酸volenasorsen,它与ApoC3的mRNA结合,使甘油三酯降低约四分之三。

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