文章：

轻链型淀粉样变性患者诊断后首年内患者报告结局的变化及其与NT-proBNP变化的关系

Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change

原文发布日期：2021-02-01

DOI: 10.1038/s41408-021-00412-8

类型: Article

开放获取: 是

英文摘要：

We conducted a prospective cohort study in newly diagnosed systemic light chain (AL) amyloidosis patients (N = 59) to study patient-reported outcomes (PROs) through the first year. The median age was 68 years with 42% female, 8% Black, and 78% lambda subtype. Organ involvement was cardiac in 66%, renal in 58%, with 25% having 3 or greater organs involved. Between baseline and 3 months, all PROMIS®-29 domain scores worsened by 0.4–4.1 points except anxiety which improved by 2.1 points. By 1 year, scores improved compared to the greatest decline at 3 months, most statistically significant for global physical health, physical function, and fatigue. On stage-adjusted survival analysis, in addition to baseline global physical and mental health, domains measuring physical function, fatigue, anxiety, depression, and social roles were associated with 1-year survival. At 1 year, PROMIS measures were associated with NT-proBNP changes and hematologic response. Among patients with an NT-proBNP response, the improvement was seen in physical function, social roles, global mental health, and anxiety. Among patients with an NT-proBNP progression, worsening was seen with anxiety, depression, sleep, and global mental health. Measuring and tracking PROs in patients with AL amyloidosis is important and these important outcomes can be used as correlative endpoints in clinical care/research.
 

摘要翻译： 

我们在新诊断的系统性轻链淀粉样变性患者（N=59）中开展了一项前瞻性队列研究，通过第一年追踪患者报告结局。患者中位年龄为68岁，42%为女性，8%为黑人，78%为λ亚型。器官受累情况为：心脏66%，肾脏58%，其中25%的患者有3个或更多器官受累。从基线到3个月期间，除焦虑评分改善2.1分外，所有PROMIS®-29领域评分均恶化0.4-4.1分。至1年时，相较于3个月时的最大衰退期，各项评分均有所改善，其中整体生理健康、生理功能和疲劳的改善具有最显著的统计学意义。经分期校正的生存分析显示，除基线整体生理和心理健康外，测量生理功能、疲劳、焦虑、抑郁和社会角色的领域均与一年生存率相关。一年时，PROMIS指标与NT-proBNP变化及血液学反应相关。在NT-proBNP有反应的患者中，生理功能、社会角色、整体心理健康和焦虑领域出现改善；而在NT-proBNP进展的患者中，焦虑、抑郁、睡眠和整体心理健康出现恶化。在AL淀粉样变性患者中测量和追踪患者报告结局具有重要意义，这些关键指标可作为临床护理/研究中的相关性终点。

原文链接：

Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change