Patient-reported outcome measures for transthyretin cardiac amyloidosis: the ITALY study.

IF 5.2 2区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Amyloid-Journal of Protein Folding Disorders Pub Date : 2024-03-01 Epub Date: 2023-09-05 DOI:10.1080/13506129.2023.2254451

Alberto Aimo, Lucio Teresi, Vincenzo Castiglione, Anna Lisa Picerni, Martina Niccolai, Silvia Severino, Assunta Agazio, Anna Carnevale Baraglia, Laura Obici, Giovanni Palladini, Lucia Ponti, Alessia Argirò, Francesco Cappelli, Federico Perfetto, Matteo Serenelli, Giancarlo Trimarchi, Roberto Licordari, Gianluca Di Bella, Olena Chubuchna, Filippo Quattrone, Sabina Nuti, Sabina De Rosis, Claudio Passino, Claudio Rapezzi, Giampaolo Merlini, Michele Emdin, Giuseppe Vergaro

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引用次数: 0

Abstract

Background: Transthyretin cardiac amyloidosis (ATTR-CA) has a deep impact on the quality of life (QoL), yet no specific patient-reported outcome measures (PROMs) for ATTR-CA exist.

Methods: The ITALY study involved 5 Italian referral centres (Pisa, Pavia, Ferrara, Florence, Messina) enrolling consecutive outpatients with ATTR-CA.

Results: Two 30-item questionnaires were created for wild-type (wt) and variant (v) ATTR-CA. Scores ranged from 100 (best condition) to 0 (worst condition). Out of 140 patients enrolled (77% with ATTRwt-CA), 115 repeated the re-evaluation at 6 months. At baseline, only 30% of patients needed help to fill out the questionnaires. Among baseline variables, all KCCQ and SF-36 domains were univariate predictors of ITALY scores in ATTRwt-CA patients, with the KCCQ Symptom Summary score (beta coefficient 0.759), Social Limitations (0.781), and Overall summary score (0.786) being the strongest predictors. The SF-36 Emotional well-being score (0.608), the KCCQ Overall summary score (0.656), and the SF-36 Energy/fatigue score (0.669) were the strongest univariate predictors of ITALY scores in ATTRv-CA. Similar results were found at 6 months.

Conclusions: The ITALY questionnaires are the first specific PROMs for ATTRwt- and ATTRv-CA. Questionnaire completion is feasible. ITALY scores display close relationships with non-ATTR-specific measures of QoL.

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转甲状腺素心脏淀粉样变性的患者报告结果衡量标准：ITALY 研究。

背景：转甲状腺素心脏淀粉样变性（ATTR-CA）对患者的生活质量（QoL）有很深的影响，但目前还没有针对ATTR-CA的患者报告结果测量指标（PROM）：ITALY研究涉及意大利的5个转诊中心（比萨、帕维亚、费拉拉、佛罗伦萨、墨西拿），连续招募门诊ATTR-CA患者：结果：针对野生型（wt）和变异型（v）ATTR-CA制作了两份共30项的问卷。得分从 100 分（最佳状态）到 0 分（最差状态）不等。在 140 名入选患者（77% 患有 ATTRwt-CA）中，115 人在 6 个月后重复了重新评估。基线时，只有 30% 的患者需要帮助填写问卷。在基线变量中，所有 KCCQ 和 SF-36 领域都是 ATTRwt-CA 患者 ITALY 评分的单变量预测因子，其中 KCCQ 症状摘要评分（β 系数为 0.759）、社交限制（0.781）和总体摘要评分（0.786）是最强的预测因子。SF-36 情绪健康评分（0.608）、KCCQ 总体汇总评分（0.656）和 SF-36 能量/疲劳评分（0.669）是 ATTRv-CA 中 ITALY 评分的最强单变量预测因子。6个月后的结果与此类似：ITALY问卷是首个针对ATTRwt-和ATTRv-CA的特定PROMs。问卷填写是可行的。ITALY 评分与非 ATTR 特异性 QoL 测量结果关系密切。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Amyloid-Journal of Protein Folding Disorders 生物-生化与分子生物学

CiteScore

10.60

自引率

10.90%

发文量

审稿时长

6-12 weeks

期刊介绍： Amyloid: the Journal of Protein Folding Disorders is dedicated to the study of all aspects of the protein groups and associated disorders that are classified as the amyloidoses as well as other disorders associated with abnormal protein folding. The journals major focus points are: etiology, pathogenesis, histopathology, chemical structure, nature of fibrillogenesis; whilst also publishing papers on the basic and chemical genetic aspects of many of these disorders. Amyloid is recognised as one of the leading publications on amyloid protein classifications and the associated disorders, as well as clinical studies on all aspects of amyloid related neurodegenerative diseases and major clinical studies on inherited amyloidosis, especially those related to transthyretin. The Journal also publishes book reviews, meeting reports, editorials, thesis abstracts, review articles and symposia in the various areas listed above.