遗传性血管性水肿生活质量问卷的内容验证和心理计量学评估
Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema.
机构信息
ICON plc, South San Francisco, CA, USA.
Takeda Development Center Americas, Inc, Lexington, MA, USA.
出版信息
J Patient Rep Outcomes. 2023 Apr 3;7(1):33. doi: 10.1186/s41687-023-00576-w.
BACKGROUND
There is considerable burden of illness in hereditary angioedema (HAE). However, instruments to assess health-related quality of life (HRQoL) in HAE are limited. The Angioedema Quality of Life Questionnaire (AE-QoL) was developed to measure HRQoL in patients with recurrent angioedema; the validity of the AE-QoL in patients with HAE is described.
METHODS
To identify disease-related experiences with a focus on the impact of HAE on HRQoL, interviews were conducted with a group of clinician experts and patients with HAE from Canada, France, Germany, Spain, the United Kingdom, and the United States, along with a targeted literature review. Concepts were mapped to the AE-QoL to assess item relevance, interpretation, and conceptual coverage. Cognitive interviews assessed item clarity and relevance. A psychometric validation was performed using data from a phase 3 trial.
RESULTS
Interviews were conducted with seven clinicians and 40 adult patients. Patients reported 35 unique impacts of HAE on their lives, the most frequent being on work/school, social relationships, physical activities, and emotions, particularly fear/worrying and anxiety. Saturation for these impacts was reached, and all concepts covered in the AE-QoL were reported during the interviews. Patients agreed that the questionnaire items and response options were clear and relevant, and the 4-week recall period was appropriate. The psychometric validation included data from 64 patients. For AE-QoL total scores, excellent internal consistency (Cronbach's alpha > 0.90), test-retest reliability (intraclass coefficient > 0.80), convergent validity with the Sheehan Disability Scale (r = 0.663), divergent validity with the EQ-5D-5L index (r = 0.292) and EQ-VAS (r = 0.337), and known-groups validity (p < 0.0001; ɳ2 = 0.56) were demonstrated.
CONCLUSIONS
Qualitative and psychometric analyses showed that the AE-QoL is a reliable and valid instrument for measuring HRQoL in adult patients with HAE from six countries.
背景
遗传性血管性水肿（HAE）患者的疾病负担较大。然而，用于评估 HAE 患者健康相关生活质量（HRQoL）的工具十分有限。Angioedema Quality of Life Questionnaire（AE-QoL）是一种用于评估复发性血管性水肿患者 HRQoL 的工具，本研究旨在评估其在 HAE 患者中的有效性。
方法
为了明确与疾病相关的体验，尤其是 HAE 对 HRQoL 的影响，本研究对来自加拿大、法国、德国、西班牙、英国和美国的一组临床医生专家和 HAE 患者进行了访谈，并进行了针对性的文献回顾。将概念映射到 AE-QoL 上，以评估其项目相关性、解释和概念涵盖范围。认知访谈评估了项目的清晰度和相关性。使用一项 3 期临床试验的数据进行了心理测量验证。
结果
本研究共进行了 7 次临床医生访谈和 40 次患者访谈。患者报告了 HAE 对其生活的 35 种不同影响，最常见的是对工作/学业、社交关系、身体活动和情绪的影响，尤其是恐惧/担忧和焦虑。这些影响都得到了充分的体现，并且在访谈中报告了 AE-QoL 涵盖的所有概念。患者一致认为问卷项目和应答选项清晰且相关，4 周的回顾期是合适的。心理测量验证包括来自 64 名患者的数据。AE-QoL 总分具有极好的内部一致性（Cronbach's alpha > 0.90）、重测信度（组内相关系数 > 0.80）、与 Sheehan 残疾量表的相关性（r = 0.663）、与 EQ-5D-5L 指数（r = 0.292）和 EQ-VAS（r = 0.337）的区分效度，以及与已知组别的相关性（p < 0.0001；ɳ2 = 0.56）。
结论
定性和心理测量分析表明，AE-QoL 是一种可靠且有效的工具，可用于评估来自六个国家的 HAE 成年患者的 HRQoL。
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