心脏淀粉样变的治疗策略进展：从发病机制的发现到诊断和治疗的进步。
Evolving Strategies in Cardiac Amyloidosis: From Mechanistic Discoveries to Diagnostic and Therapeutic Advances.
机构信息
Division of Cardiology, Department of Medicine, University of California, Los Angeles, Los Angeles, CA, USA; UCLA Cardio-Oncology Program, Division of Cardiology, Department of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.
Department of Medicine, University of California, Los Angeles, Los Angeles, CA, USA.
出版信息
Cardiol Clin. 2025 Feb;43(1):93-110. doi: 10.1016/j.ccl.2024.09.006. Epub 2024 Oct 22.
Diagnosis and treatment of cardiac amyloidosis have rapidly evolved over the past decade by harnessing mechanisms of disease pathogenesis. Cardiac amyloidosis is caused by myocardial deposition of fibrils formed by misfolded proteins, namely transthyretin (ATTR) and immunoglobulin light chains (AL). Advances in noninvasive imaging have revolutionized diagnosis of ATTR cardiomyopathy (CM). Novel treatments for ATTR-CM utilize a range of therapeutic techniques, including protein stabilizers, interfering RNA, gene editing, and monoclonal antibodies. AL-CM, primarily driven by plasma cell dyscrasias, requires treatment with chemotherapy and consideration for autologous stem cell transplant. These incredible advances aim to improve patient outcomes in cardiac amyloidosis.
在过去的十年中，通过利用疾病发病机制的机制，心脏淀粉样变性的诊断和治疗迅速发展。心脏淀粉样变性是由错误折叠的蛋白质（即转甲状腺素蛋白（ATTR）和免疫球蛋白轻链（AL））形成的纤维在心肌中的沉积引起的。非侵入性成像技术的进步彻底改变了ATTR 心肌病（CM）的诊断。用于治疗ATTR-CM 的新型治疗方法采用了一系列治疗技术，包括蛋白质稳定剂、干扰 RNA、基因编辑和单克隆抗体。AL-CM 主要由浆细胞异常引起，需要接受化疗，并考虑自体干细胞移植。这些令人难以置信的进展旨在改善心脏淀粉样变性患者的预后。
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