Development and validation of a US quality of life instrument for hereditary angioedema due to C1 inhibitor deficiency

IF 5.8 2区医学 Q1 ALLERGY

Annals of Allergy Asthma & Immunology Pub Date : 2024-12-01 DOI:10.1016/j.anai.2024.07.018

Anthony J. Castaldo MPA , Nikolaj Siersbæk PhD , Deborah Corcoran MSc , Christine N. Selva BS , Christian Jervelund MSc , Sandra C. Christiansen MD , Marc A. Riedl MD , Bruce L. Zuraw MD , Paula J. Busse MD

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引用次数: 0

Abstract

Background

Hereditary angioedema (HAE) attacks are unpredictable, cause a substantial and enduring burden of illness, and are potentially fatal. Because of issues unique to the US health care system, there is a need for a US-validated, HAE-specific quality of life (QoL) instrument.

Objective

To develop and validate a US HAE-specific QoL instrument according to US Food and Drug Administration guidelines and established methodologies.

Methods

We generated 41 QoL-related items likely relevant to US patients with HAE due to C1 inhibitor (C1INH) deficiency (HAE-C1INH) and performed a 10-patient pilot study to refine the question wording. A total of 415 US patients with HAE-C1INH completed the initial 41-item instrument online, thereby providing data for item reduction, factor analysis, and the assessment of validity and reliability. We used a multiple linear regression to identify the drivers of the total and domain scores. Convergent validity analysis was used to assess the extent to which the HAE-C1INH QoL instrument (HAE-C1INH-QoL) is theoretically related to the angioedema-QoL instrument (AE-QoL).

Results

Item reduction and factor analysis yielded a final instrument of 31 items across 5 domains, and the assessment analysis showed that the HAE-C1INH-QoL is valid and reliable. Attack frequency and severity were statistically significant factors that influenced the total and domain scores. Correlation analysis of the 2 instruments indicated that 8 items of the HAE-C1INH-QoL were not included or well-described in the AE-QoL.

Conclusion

The HAE-C1INH-QoL is the first HAE-specific QoL tool validated in the United States. When compared with the AE-QoL, the items in our instrument are more relevant to US patients with HAE.

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美国 HAE-C1INH 生活质量工具的开发与验证。

背景：遗传性血管性水肿（HAE）的发作难以预测，会造成巨大而持久的疾病负担，并可能致命。由于美国医疗系统的特殊性，需要一种经过美国验证的专门针对 HAE 的生活质量（QoL）工具：按照美国食品药品管理局的指导方针和既定方法，开发并验证美国 HAE 专用生活质量工具：我们制定了可能与美国 HAE-C1INH 患者相关的 41 个 QoL 相关项目，并对 10 名患者进行了试点研究，以完善问题措辞。415 名缺乏 C1 抑制剂 (C1INH) 的美国 HAE 患者在线完成了最初的 41 个项目的问卷调查，为项目缩减、因素分析以及有效性和可靠性评估提供了数据。我们使用多元线性回归来确定总分和领域得分的驱动因素。收敛有效性分析评估了 HAE-C1INH-QoL 与血管性水肿-QoL（AE-QoL）的理论相关程度：结果：通过缩减项目和因子分析，最终形成了由 31 个项目组成、涵盖 5 个领域的问卷，评估分析表明 HAE-C1INH-QoL 具有有效性和可靠性。在统计学上，发作频率和严重程度是影响总分和领域得分的重要因素。两种工具的相关性分析表明，HAE-C1INH-QoL中有8个项目未包含在AE-QoL中或未在AE-QoL中得到很好的描述：结论：HAE-C1INH-QoL 是首个在美国通过验证的 HAE 专属 QoL 工具。结论：HAE-C1INH-QoL 是首个在美国通过验证的 HAE 专属 QoL 工具。与 AE-QoL 相比，我们的工具中的项目更适合美国 HAE 患者。

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

Annals of Allergy Asthma & Immunology 医学-过敏

CiteScore

6.50

自引率

6.80%

发文量

437

审稿时长

33 days

期刊介绍： Annals of Allergy, Asthma & Immunology is a scholarly medical journal published monthly by the American College of Allergy, Asthma & Immunology. The purpose of Annals is to serve as an objective evidence-based forum for the allergy/immunology specialist to keep up to date on current clinical science (both research and practice-based) in the fields of allergy, asthma, and immunology. The emphasis of the journal will be to provide clinical and research information that is readily applicable to both the clinician and the researcher. Each issue of the Annals shall also provide opportunities to participate in accredited continuing medical education activities to enhance overall clinical proficiency.