Division of Hematology, Bnai Zion Medical Centre, Haifa, Israel.
The Ruth and Bruce Rappaport Faculty of Medicine, Technion, Haifa, Israel.
Expert Rev Hematol. 2023 Jan;16(1):33-49. doi: 10.1080/17474086.2023.2166924. Epub 2023 Jan 12.
Systemic AL amyloidosis (ALA) is a clonal plasma cell (PC) disease characterized by deposition of amyloid fibrils in different organs and tissues. Traditionally, the prognosis of ALA is poor and is primarily defined by cardiac involvement. The modern prognostic models are based on cardiac markers and free light chain difference (dFLC). Cardiac biomarkers have low specificity and are dependent on renal function, volume status, and cardiac diseases other than ALA. New therapies significantly improved the prognosis of the disease. The advancements in technologies - cardiac echocardiography (ECHO) and cardiac MRI (CMR), as well as new biological markers, relying on cardiac injury, inflammation, endothelial damage, and clonal and non-clonal PC markers are promising.
An update on the prognostic significance of cardiac ALA, number of involved organs, response to treatment, including minimal residual disease (MRD), ECHO, MRI, and new biological markers will be discussed. The literature search was done in PubMed and Google Scholar, and the most recent and relevant data are included.
Prospective multicenter trials, evaluating multiple clinical and laboratory parameters, should be done to improve the risk assessment models in ALA in the modern era of therapy.
系统性 AL 淀粉样变性（ALA）是一种克隆性浆细胞（PC）疾病，其特征是在不同的器官和组织中沉积淀粉样纤维。传统上，ALA 的预后较差，主要由心脏受累定义。现代预后模型基于心脏标志物和游离轻链差（dFLC）。心脏生物标志物特异性低，且依赖于肾功能、容量状态以及除 ALA 以外的心脏疾病。新疗法显著改善了疾病的预后。技术的进步——心脏超声心动图（ECHO）和心脏 MRI（CMR），以及依赖于心脏损伤、炎症、内皮损伤以及克隆和非克隆 PC 标志物的新型生物标志物，都具有广阔的应用前景。
本文将讨论心脏 ALA 的预后意义、受累器官数量、治疗反应，包括微小残留疾病（MRD）、ECHO、MRI 和新型生物标志物的最新进展。文献检索在 PubMed 和 Google Scholar 中进行，纳入了最新和最相关的数据。
应开展前瞻性多中心试验，评估多个临床和实验室参数，以在治疗的现代时代改进 ALA 的风险评估模型。
