Light-chain amyloidosis (AL) is a rare multisystem disorder characterized by the deposition of misfolded amyloid fibrils derived from monoclonal immunoglobulin light chains in various organs. One of the most common organs involved in AL is the heart, with 50–70% of patients clinically symptomatic at diagnosis. We conducted a multi-center, retrospective analysis of 67 patients diagnosed between July 2012 and August 2022 with the European 2012 modification of Mayo 2004 stage III cardiac AL. The most important factors identified in the univariate Cox analysis contributing to a longer OS included Eastern Cooperative Oncology Group performance status (ECOG PS) ≤ 1, New York Heart Association functional classification (NYHA FC) ≤ 2, the use of autologous stem cell transplantation (ASCT) after induction treatment, achieving a hematological response (≥very good partial response) and cardiac (≥partial response) response after first-line treatment. The most important prognostic factors with the most significant impact on OS improvement in patients with modified Mayo stage III cardiac AL identified by multivariate Cox analysis are ECOG PS ≤ 1, NYHA FC ≤ 2, and achieving hematological response ≥ VGPR and cardiac response ≥ PR after first-line treatment.
轻链型淀粉样变性（AL）是一种罕见的全身性疾病，其特征是由单克隆免疫球蛋白轻链衍生的错误折叠淀粉样纤维在不同器官中沉积。心脏是AL最常累及的器官之一，50-70%的患者在诊断时已出现临床症状。我们对2012年7月至2022年8月期间确诊的67例采用欧洲2012年修订版Mayo 2004 III期心脏AL分期的患者进行了多中心回顾性分析。单变量Cox分析显示，与较长总生存期相关的最重要因素包括：美国东部肿瘤协作组体能状态评分（ECOG PS）≤1、纽约心脏病协会心功能分级（NYHA FC）≤2、诱导治疗后接受自体干细胞移植（ASCT）、一线治疗后达到血液学缓解（≥非常好的部分缓解）及心脏缓解（≥部分缓解）。多变量Cox分析进一步确定，对于修订版Mayo III期心脏AL患者，对总生存期改善最具显著影响的预后因素为：ECOG PS≤1、NYHA FC≤2，以及一线治疗后达到血液学缓解≥VGPR和心脏缓解≥PR。
肿瘤(癌症)患者之家