Cianci Vincenzo, Cianci Alessio, Sapienza Daniela, Cracò Annalisa, Germanà Antonino, Ieni Antonio, Gualniera Patrizia, Asmundo Alessio, Mondello Cristina
Department of Biomedical and Dental Sciences and Morphofunctional Imaging, Section of Legal Medicine, University of Messina, Via Consolare Valeria, 1, 98125 Messina, Italy.
Department of Cardiovascular Medicine, Fondazione Policlinico Universitario A. Gemelli-IRCCS, Largo A. Gemelli 8, 00168 Rome, Italy.
J Clin Med. 2024 Aug 29;13(17):5140. doi: 10.3390/jcm13175140.
Cardiac amyloidosis is an infiltrative disease that causes progressive myocardial impairment secondary to amyloid fibril deposition in the extracellular space of the myocardium. Many amyloid precursors, including transthyretin protein, are known to determine cardiac damage by aggregating and precipitating in cardiac tissue. Transthyretin cardiac amyloidosis may be either caused by rare genetic mutations of the transthyretin gene in the hereditary variant, or may arise as a consequence of age-related mechanisms in the acquired form. Although it has been labeled as a rare disease, in recent years, transthyretin cardiac amyloidosis has stood out as an emerging cause of aortic stenosis, unexplained left ventricular hypertrophy and heart failure with preserved ejection fraction, particularly in the elderly. Indeed, the integration of data deriving from both in vivo imaging techniques (whose advancement in the last years has allowed to achieve an easier and more accessible non-invasive diagnosis) and forensic studies (showing a prevalence of amyloid deposition in cardiac tissue of elderly patients up to 29%) suggests that cardiac amyloidosis is a more common disease than traditionally considered. Thanks to all the improvements in non-invasive diagnostic techniques, along with the development of efficacious therapies offering improvements in survival rates, transthyretin cardiac amyloidosis has been transformed from an incurable and infrequent condition to a relatively more diffuse and treatable disease, which physicians should take into consideration in the differential diagnostic processes in daily clinical practice.
心脏淀粉样变性是一种浸润性疾病,由于淀粉样原纤维沉积在心肌细胞外间隙而导致进行性心肌损害。已知许多淀粉样前体,包括转甲状腺素蛋白,通过在心脏组织中聚集和沉淀来决定心脏损伤。转甲状腺素蛋白心脏淀粉样变性可能由遗传性变体中转甲状腺素基因的罕见基因突变引起,也可能是获得性形式中与年龄相关机制的结果。尽管它被标记为罕见疾病,但近年来,转甲状腺素蛋白心脏淀粉样变性已成为主动脉瓣狭窄、不明原因左心室肥厚和射血分数保留的心力衰竭的一个新出现的病因,尤其是在老年人中。事实上,来自体内成像技术(近年来其进步使得能够实现更简便、更易获得的非侵入性诊断)和法医研究(显示老年患者心脏组织中淀粉样沉积的患病率高达29%)的数据整合表明,心脏淀粉样变性是一种比传统认为更常见的疾病。由于非侵入性诊断技术的所有改进,以及有效疗法的发展使生存率得到提高,转甲状腺素蛋白心脏淀粉样变性已从一种无法治愈且罕见的疾病转变为一种相对更普遍且可治疗的疾病,医生在日常临床实践的鉴别诊断过程中应予以考虑。