文章：

AL型淀粉样变性生存率的显著改善：一项40年纵向自然病史研究

Marked progress in AL amyloidosis survival: a 40-year longitudinal natural history study

原文发布日期：2021-08-04

DOI: 10.1038/s41408-021-00529-w

类型: Article

开放获取: 是

英文摘要：

The recent decades have ushered in considerable advancements in the diagnosis and treatment of systemic light chain (AL) amyloidosis. As disease outcomes improve, AL amyloidosis-unrelated factors may impact mortality. In this study, we evaluated survival trends and primary causes of death among 2337 individuals with AL amyloidosis referred to the Boston University Amyloidosis Center. Outcomes were analyzed according to date of diagnosis: 1980-1989 (era 1), 1990-1999 (era 2), 2000-2009 (era 3), and 2010-2019 (era 4). Overall survival increased steadily with median values of 1.4, 2.6, 3.3, and 4.6 years for eras 1–4, respectively (P < 0.001). Six-month mortality decreased over time from 23% to 13%. Wide gaps in survival persisted amid patient subgroups; those with age at diagnosis ≥70 years had marginal improvements over time. Most deaths were attributable to disease-related factors, with cardiac failure (32%) and sudden unexpected death (23%) being the leading causes. AL amyloidosis-unrelated mortality increased across eras (from 3% to 16% of deaths) and with longer-term survival (29% of deaths occurring >10 years after diagnosis). Under changing standards of care, survival improved and early mortality declined over the last 40 years. These findings support a more optimistic outlook for patients with AL amyloidosis.
 

摘要翻译： 

近几十年来，系统性轻链（AL）型淀粉样变性的诊断与治疗取得了显著进展。随着疾病预后的改善，非AL型淀粉样变性相关因素可能对死亡率产生影响。本研究评估了波士顿大学淀粉样变性中心2337例AL型淀粉样变性患者的生存趋势及主要死亡原因。我们根据诊断年份进行分析：1980-1989年（时期1）、1990-1999年（时期2）、2000-2009年（时期3）和2010-2019年（时期4）。总生存期稳步提升，时期1至4的中位生存期分别为1.4年、2.6年、3.3年和4.6年（P < 0.001）。6个月死亡率从23%下降至13%。患者亚组间的生存差距仍然显著；诊断年龄≥70岁的患者随时间推移仅获得有限改善。多数死亡与疾病相关因素有关，其中心力衰竭（32%）和突发意外死亡（23%）是主要死因。非AL型淀粉样变性相关死亡率随时代推移（从3%增至16%）及长期生存者增加而上升（诊断10年后的死亡中占29%）。在过去40年间，随着诊疗标准的变迁，患者生存率得到提高，早期死亡率显著下降。这些发现为AL型淀粉样变性患者提供了更为乐观的预后展望。

原文链接：

Marked progress in AL amyloidosis survival: a 40-year longitudinal natural history study