Medarametla Gnana Deepthi, Kahlon Ripudaman Singh, Mahitha Lampimukhi, Shariff Sanobar, Vakkalagadda Naga Praneeth, Chopra Hitesh, Kamal Mohammad Amjad, Patel Neil, Sethi Yashendra, Kaka Nirja
Pranavi Children's and Eye Hospital, Kandukuru, Andhra Pradesh, India.
Pear Research, Dehradun, India.
EXCLI J. 2023 Aug 3;22:781-808. doi: 10.17179/excli2023-6284. eCollection 2023.
Amyloidosis is a protein deposition disorder in which insoluble fibril structures accumulate in the bodily tissues damaging the organ function. Cardiac amyloidosis is a severe but under-reported medical condition characterized by the accumulation of amyloid in the extracellular area of the myocardium, which results in thickening and stiffening of ventricular walls. Cardiac amyloidosis has recently gained much attention with its slowly surging incidence. With this study, we seek to comprehensively compile the pathophysiology and clinical picture of cardiac amyloidosis subtypes, extending a clinically oriented, up-to-date clinical approach to diagnosis and therapy. Cardiac amyloidosis can be caused by rare genetic mutations which may be inherited or acquired. The growing incidence can be attributed to advancements in imaging methods and other diagnostic modalities. Most occurrences of cardiac amyloidosis result from two forms of precursor protein: transthyretin [TTR] amyloid and immunoglobulin-derived light-chain amyloid. Prompt identification of cardiac amyloidosis can facilitate the implementation of evolving therapeutic interventions to enhance the outcomes. The modalities for the management of CA have evolved significantly in the last ten years. Apart from therapies for modifying disease and heart failure, a myriad of novel therapeutic approaches that target specific aspects of the disease, including gene therapies, are being researched. These aim at impeding its progression and improving clinical outcomes. See also Figure 1(Fig. 1).
淀粉样变性是一种蛋白质沉积性疾病,其中不溶性纤维结构在身体组织中积聚,损害器官功能。心脏淀粉样变性是一种严重但报告不足的病症,其特征是淀粉样蛋白在心肌细胞外区域积聚,导致心室壁增厚和僵硬。随着其发病率的缓慢上升,心脏淀粉样变性最近受到了广泛关注。通过这项研究,我们旨在全面汇编心脏淀粉样变性亚型的病理生理学和临床表现,扩展一种以临床为导向的、最新的诊断和治疗临床方法。心脏淀粉样变性可能由罕见的基因突变引起,这些突变可能是遗传的或后天获得的。发病率的上升可归因于成像方法和其他诊断方式的进步。大多数心脏淀粉样变性病例由两种形式的前体蛋白引起:转甲状腺素蛋白(TTR)淀粉样蛋白和免疫球蛋白衍生的轻链淀粉样蛋白。及时识别心脏淀粉样变性有助于实施不断发展的治疗干预措施,以改善治疗效果。在过去十年中,心脏淀粉样变性的管理方式有了显著发展。除了针对疾病和心力衰竭的治疗方法外,正在研究许多针对该疾病特定方面的新型治疗方法,包括基因治疗。这些旨在阻止其进展并改善临床结果。另见图1(图1)。