a Amyloidosis Research and Treatment Center, Foundation 'Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Policlinico San Matteo', and Department of Molecular Medicine , University of Pavia , Pavia , Italy.
b PhD program in Experimental Medicine , University of Pavia , Pavia , Italy.
Expert Rev Hematol. 2018 Feb;11(2):117-127. doi: 10.1080/17474086.2018.1424534. Epub 2018 Jan 10.
The most common form of systemic amyloidosis in Western countries is light chain amyloidosis. It is characterized by the deposition of a misfolded light chain in target organs. This amyloid precursor is produced by a usually small but dangerous B-cell clone. Areas covered: This review examines the diagnostic workup of this disease and current knowledge of biomarker-based staging systems. In addition, a risk-adapted treatment approach is presented, as well as an overview of the new treatment strategies. Expert commentary: The cornerstone of treatment is rapid and effective chemotherapy targeting the underlying plasma cell clone. In the near future, this will probably be associated with novel approaches targeting other steps of the amyloid cascade that result in amyloid deposits. Currently available effective treatments can alter its natural history if an early diagnosis is made. The availability of novel, more powerful drugs, and identification of the cellular mechanisms of organ damage and of the characteristics of the amyloidogenic plasma-cell clone all give grounds to hope that a dramatic improvement in the treatment of this disease will be seen in the near future.
西方国家最常见的系统性淀粉样变是轻链淀粉样变。其特征是在靶器官中沉积错误折叠的轻链。这种淀粉样前体由通常较小但危险的 B 细胞克隆产生。涵盖领域：本篇综述检查了该疾病的诊断方法和基于生物标志物的分期系统的现有知识。此外，还提出了一种风险适应的治疗方法，并概述了新的治疗策略。专家评论：治疗的基石是针对潜在浆细胞克隆的快速有效的化疗。在不久的将来，这可能会与靶向导致淀粉样沉积的淀粉样蛋白级联的其他步骤的新方法相关联。如果早期诊断，目前可用的有效治疗方法可以改变其自然病史。新型、更有效的药物的出现，以及对器官损伤的细胞机制和淀粉样浆细胞克隆特征的鉴定，都使人们有理由希望在不久的将来，这种疾病的治疗会有显著改善。
