Cardiology Division, University of Pisa, Italy.
Haematology Department, University of Pisa, Italy.
Int J Cardiol. 2018 Nov 15;271:152-160. doi: 10.1016/j.ijcard.2018.05.018.
Light-chain (AL) amyloidosis is the most common type of systemic amyloidosis, affecting around 10 people per million per year. This serious disorder is characterized by the presence of a clone of bone marrow plasma cells that produces monoclonal light chains (LCs) of the κ or predominantly λ type. These amyloidogenic LCs undergo extracellular misfolding and aggregation into proteotoxic soluble oligomers and amyloid fibrils that deposit within tissues. The lethal consequences of AL amyloidosis are due to the toxic products (the LCs) and not to the malignant behaviour of the plasma cell clone. Almost 80% of patients with AL amyloidosis have some degree of cardiac involvement, manifesting as heart failure (HF), and carrying a particularly poor prognosis. The past decade has seen major advances in the treatment of AL amyloidosis, and a rapidly fatal disease has become a treatable and possibly curable condition. The number of therapeutic options is rapidly expanding, offering hope to address currently unmet needs (most notably, the treatment of frail patients). The treatment of AL amyloidosis consists in a combination of agents targeting multiple steps of the amyloid cascade, associated with effective HF management, and there is ground for hope for dramatically improving the outcome in the near future. In the present review we will summarize our current knowledge on therapy for cardiac AL amyloidosis, targeting clinical cardiologists involved in the care of this serious disorder.
轻链 (AL) 淀粉样变是最常见的系统性淀粉样变，每年每百万人中有 10 人左右受影响。这种严重的疾病的特征是存在骨髓浆细胞的克隆，该克隆产生κ或主要为λ型的单克隆轻链 (LC)。这些淀粉样变性的 LC 经历细胞外错误折叠和聚集到毒性可溶性寡聚体和淀粉样纤维中，在组织中沉积。AL 淀粉样变的致命后果是由于毒性产物（LC）而不是浆细胞克隆的恶性行为。几乎 80%的 AL 淀粉样变患者存在一定程度的心脏受累，表现为心力衰竭 (HF)，预后特别差。过去十年中，AL 淀粉样变的治疗取得了重大进展，一种迅速致命的疾病已成为可治疗且可能治愈的疾病。治疗选择的数量正在迅速增加，为解决当前未满足的需求（尤其是脆弱患者的治疗）带来了希望。AL 淀粉样变的治疗包括针对淀粉样蛋白级联的多个步骤的联合用药，以及有效的 HF 管理，有望在不久的将来显著改善预后。在本综述中，我们将总结我们目前对心脏 AL 淀粉样变治疗的认识，针对涉及这种严重疾病治疗的临床心脏病专家。
