轻链淀粉样变性的功能状态和生活质量：先进的成像技术、纵向变化和结果。

IF 10.3 1区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS

JACC. Heart failure Pub Date : 2024-12-01 DOI:10.1016/j.jchf.2024.07.007

Olivier F. Clerc MD, MPH , Shilpa Vijayakumar MD, MPH , Sarah A.M. Cuddy MD , Giada Bianchi MD , Jocelyn Canseco Neri MS , Alexandra Taylor BS , Dominik C. Benz MD , Yesh Datar BA , Marie Foley Kijewski PhD , Andrew J. Yee MD , Frederick L. Ruberg MD , Ronglih Liao PhD , Rodney H. Falk MD , Vaishali Sanchorawala MD , Sharmila Dorbala MD, MPH

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引用次数: 0

摘要

背景：在轻链（AL）淀粉样变性中，功能状态和心力衰竭相关生活质量（HF-QOL）是否与心肌病严重程度相关、是否随治疗而改善、是否与主要心脏不良事件（MACE）相关（超出有效评分），目前尚不清楚：作者旨在作者旨在：1）将功能状态和 HF-QOL 与心肌病严重程度相关联；2）分析它们的纵向变化；3）评估它们与 MACE 的独立关联：这项研究纳入了106名AL淀粉样变性患者，其中81%患有AL心肌病。采用 NYHA 功能分级、Karnofsky 量表和 6 分钟步行距离（6MWD）评估功能状态，采用明尼苏达心衰患者生活问卷（MLWHFQ）评估高频-QOL。心肌病的严重程度通过心脏18F-氟贝他哌正电子发射断层扫描/计算机断层扫描、心脏磁共振、超声心动图和血清心脏生物标记物进行评估。MACE定义为全因死亡、心衰住院或心脏移植：新近确诊的 AL 型心肌病患者的 NYHA 功能分级、Karnofsky 评分、6MWD 和 MLWHFQ 严重受损（P < 0.001），并与心肌病严重程度的所有指标相关（P ≤ 0.010）。心肌病患者的 NYHA 功能分级、6MWD 和 MLWHFQ 在 12 个月时有所改善（P ≤ 0.013）。所有功能状态和HF-QOL指标均与MACE相关（P≤ 0.017），6MWD和MLWHFQ指标与梅奥分期无关（P≤ 0.006）：结论：功能状态和高频-QOL与AL型心肌病的严重程度相关，在12个月内接受治疗后会得到改善，并且与MACE相关，与6MWD和MLWHFQ的梅奥分期无关。除预后评分外，这些指标还可作为未来研究的替代结果进行进一步验证。

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Functional Status and Quality of Life in Light-Chain Amyloidosis

Background

In light-chain (AL) amyloidosis, whether functional status and heart failure–related quality of life (HF-QOL) correlate with cardiomyopathy severity, improve with therapy, and are associated with major adverse cardiac events (MACE) beyond validated scores is not well-known.

Objectives

The authors aimed to: 1) correlate functional status and HF-QOL with cardiomyopathy severity; 2) analyze their longitudinal changes; and 3) assess their independent associations with MACE.

Methods

This study included 106 participants with AL amyloidosis, with 81% having AL cardiomyopathy. Functional status was evaluated using the NYHA functional class, the Karnofsky scale, and the 6-minute walk distance (6MWD), and HF-QOL using the MLWHFQ (Minnesota Living with Heart Failure Questionnaire). Cardiomyopathy severity was assessed by cardiac ¹⁸F-florbetapir positron emission tomography/computed tomography, cardiac magnetic resonance, echocardiography, and serum cardiac biomarkers. MACE were defined as all-cause death, heart failure hospitalization, or cardiac transplantation.

Results

NYHA functional class, Karnofsky scale, 6MWD, and MLWHFQ were impaired substantially in participants with recently diagnosed AL cardiomyopathy (P < 0.001), and correlated with all markers of cardiomyopathy severity (P ≤ 0.010). NYHA functional class, 6MWD, and MLWHFQ improved at 12 months in participants with cardiomyopathy (P ≤ 0.013). All measures of functional status and HF-QOL were associated with MACE (P ≤ 0.017), independent of Mayo stage for 6MWD and MLWHFQ (P ≤ 0.006).

Conclusions

Functional status and HF-QOL were associated with AL cardiomyopathy severity, improved on therapy within 12 months, and were associated with MACE, independently of Mayo stage for 6MWD and MLWHFQ. They may be validated further in addition to prognostic scores and as surrogate outcomes for future studies.

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来源期刊

JACC. Heart failure CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

21.20

自引率

2.30%

发文量

164

期刊介绍： JACC: Heart Failure publishes crucial findings on the pathophysiology, diagnosis, treatment, and care of heart failure patients. The goal is to enhance understanding through timely scientific communication on disease, clinical trials, outcomes, and therapeutic advances. The Journal fosters interdisciplinary connections with neuroscience, pulmonary medicine, nephrology, electrophysiology, and surgery related to heart failure. It also covers articles on pharmacogenetics, biomarkers, and metabolomics.