Lovley Andrew, Hsu Kristen, LaGasse Kaitlin, Lousada Isabelle, McCausland Kristen L, Carty Michelle K, Rebello Sabrina, Bjorner Jakob B
IQVIA Quality Metric, 225 Dyer St, 2nd Floor, Providence, RI, 02903, USA.
Amyloidosis Research Consortium, 320 Nevada Street, Suite 210, Newton, MA, 02460, USA.
J Patient Rep Outcomes. 2025 Apr 29;9(1):44. doi: 10.1186/s41687-025-00880-7.
The diversity of disease phenotypes associated with transthyretin (ATTR) amyloidosis poses challenges for measurement of health outcomes. The Transthyretin Amyloidosis - Quality of Life (ATTR-QOL) Questionnaire is a disease-specific patient-reported outcome (PRO) measure of the symptoms and impacts of ATTR amyloidosis. The objective of this study was to evaluate the structural validity, reliability, and construct validity of the ATTR-QOL Impact scales.
This was a non-interventional, online survey study of adults with self-reported diagnosis of symptomatic ATTR amyloidosis. The survey included the ATTR-QOL and additional criterion measures. A scoring algorithm was proposed and tested. Factor structure, differential item functioning, and psychometric properties were explored.
The analytic sample included 233 patients. Satisfactory fit was found for a 4-factor model of disease impacts (including Daily Activities, Physical Functioning, Social/Role Functioning, and Emotional Wellbeing) and a scoring algorithm was developed. Twelve impact items were dropped from the ATTR-QOLv2 as a result of factor and differential item functioning analyses. Each scale showed evidence of satisfactory internal consistency reliability (Cronbach's α range = 0.85-0.97) and test-retest reliability at 1 week (intraclass coefficient range = 0.84-0.97). Convergent validity was supported by correlations ≥ 0.30 between ATTR-QOL Impact scale scores and other PRO measures of related constructs. The ATTR-QOL Impact scales showed greater impact among patients with worse symptom severity, cardiac functioning, or unemployment due to ATTR amyloidosis (all p < 0.05), supporting known-groups validity.
The ATTR-QOL is a reliable and valid measure of impacts meaningful to patients with ATTR amyloidosis. This study resulted in updates to the ATTR-QOL for item reduction and the development of a scoring algorithm. Ongoing studies are collecting data to evaluate the symptom scales of the ATTR-QOL.
与转甲状腺素蛋白（ATTR）淀粉样变性相关的疾病表型多样性给健康结果的测量带来了挑战。转甲状腺素蛋白淀粉样变性-生活质量（ATTR-QOL）问卷是一种针对疾病的患者报告结局（PRO）指标，用于衡量ATTR淀粉样变性的症状和影响。本研究的目的是评估ATTR-QOL影响量表的结构效度、信度和构想效度。
这是一项针对自我报告有症状性ATTR淀粉样变性诊断的成年人的非干预性在线调查研究。该调查包括ATTR-QOL和其他标准测量指标。提出并测试了一种评分算法。探索了因子结构、项目功能差异和心理测量特性。
分析样本包括233名患者。发现疾病影响的四因子模型（包括日常活动、身体功能、社会/角色功能和情绪健康）拟合良好，并开发了一种评分算法。由于因子和项目功能差异分析，12个影响项目从ATTR-QOLv2中删除。每个量表都显示出令人满意的内部一致性信度（Cronbach's α范围=0.85-0.97）和1周时的重测信度（组内相关系数范围=0.84-0.97）。ATTR-QOL影响量表得分与相关构想的其他PRO指标之间的相关性≥0.30，支持了收敛效度。ATTR-QOL影响量表在症状严重程度、心脏功能较差或因ATTR淀粉样变性失业的患者中显示出更大的影响（所有p<0.05），支持已知群体效度。
ATTR-QOL是一种可靠且有效的指标，可衡量对ATTR淀粉样变性患者有意义的影响。本研究对ATTR-QOL进行了更新，以减少项目数量并开发了一种评分算法。正在进行的研究正在收集数据以评估ATTR-QOL的症状量表。
