Blaser Mark C, Bäck Magnus, Lüscher Thomas F, Aikawa Elena
Center for Interdisciplinary Cardiovascular Sciences, Brigham and Women's Hospital, Harvard Medical School, 3 Blackfan Street, 17th Floor, Boston, MA 02115, USA.
Department of Medicine, Center for Molecular Medicine, Karolinska Institutet, Stockholm, Sweden.
Eur Heart J. 2025 Feb 14;46(7):620-634. doi: 10.1093/eurheartj/ehae829.
Calcific aortic valve disease (CAVD) resulting in aortic stenosis (AS) is the most common form of valvular heart disease, affecting 2% of those over age 65. Those who develop symptomatic severe AS have an average further lifespan of <2 years without valve replacement, and three-quarters of these patients will develop heart failure, undergo valve replacement, or die within 5 years. There are no approved pharmaceutical therapies for AS, due primarily to a limited understanding of the molecular mechanisms that direct CAVD progression in the complex haemodynamic environment. Here, advances in efforts to understand the pathogenesis of CAVD and to identify putative drug targets derived from recent multi-omics studies [including (epi)genomics, transcriptomics, proteomics, and metabolomics] of blood and valvular tissues are reviewed. The recent explosion of single-cell omics-based studies in CAVD and the pathobiological and potential drug discovery insights gained from the application of omics to this disease area are a primary focus. Lastly, the translation of knowledge gained in valvular pathobiology into clinical therapies is addressed, with a particular emphasis on treatment regimens that consider sex-specific, renal, and lipid-mediated contributors to CAVD, and ongoing Phase I/II/III trials aimed at the prevention/treatment of AS are described.
导致主动脉瓣狭窄(AS)的钙化性主动脉瓣疾病(CAVD)是最常见的心脏瓣膜病形式,影响着2%的65岁以上人群。出现症状性重度AS的患者,如果不进行瓣膜置换,平均剩余寿命不足2年,其中四分之三的患者会在5年内出现心力衰竭、接受瓣膜置换或死亡。目前尚无获批用于治疗AS的药物疗法,这主要是因为在复杂的血流动力学环境中,人们对指导CAVD进展的分子机制了解有限。在此,本文综述了在理解CAVD发病机制以及从近期血液和瓣膜组织的多组学研究[包括(表观)基因组学、转录组学、蛋白质组学和代谢组学]中确定潜在药物靶点方面所取得的进展。基于单细胞组学的CAVD研究近期激增,以及将组学应用于该疾病领域所获得的病理生物学和潜在药物发现见解是主要关注点。最后,探讨了瓣膜病理生物学中获得的知识向临床治疗的转化,特别强调了考虑性别特异性、肾脏和脂质介导的CAVD影响因素的治疗方案,并描述了旨在预防/治疗AS的正在进行的I/II/III期试验。
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