Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema

Abstract

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.

CRUSE®—An innovative mobile application for patient monitoring and management in chronic spontaneous urticaria

“…CRUSE ® comprises validated urticaria PROMs to assess disease activity and control, namely the urticaria activity score (UAS), 18,19 angioedema activity score (AAS), 20 urticaria control test (UCT), 21,22 and angioedema control test (AECT 23,24,36,37 ; see Table 1 for details). It also includes the EQ‐5D visual analogue scale (EQ‐5D VAS) 38 . This quantitative measure reflects the patients' judgement on how their health is on that day, ranging from ‘the best health you can imagine’ to ‘the worst health you can imagine’.…”

“…It also includes the EQ‐5D visual analogue scale (EQ‐5D VAS). 38 This quantitative measure reflects the patients' judgement on how their health is on that day, ranging from ‘the best health you can imagine’ to ‘the worst health you can imagine’. PROM data are collected daily for UAS, AAS, urticaria/angioedema impact on work or school performance (assessed using VAS); they are collected monthly for UCT and AECT (Figure 1 ).…”

Clinical Characteristics and Quality of Life in a Cohort of Polish Pediatric Patients with Hereditary Angioedema

“…According to studies conducted on adult patients, HAE interferes with everyday activities, limits work or school productivity, and extends to patients' leisure time. The burden of HAE affects the time free from attacks, as patients report depression and worry about the future course of the disease, uncertainty about the timing of attacks, and inheritance risk in children [7]. Instruments specifically designed to assess HRQoL in HAE that are validated include the "Hereditary Angioedema Quality of Life Questionnaire" [8], the "HAE Patient-Reported Outcomes Questionnaire" [9], and the "United States HAE Association QoL Questionnaire" [10].…”

“…Instruments specifically designed to assess HRQoL in HAE that are validated include the "Hereditary Angioedema Quality of Life Questionnaire" [8], the "HAE Patient-Reported Outcomes Questionnaire" [9], and the "United States HAE Association QoL Questionnaire" [10]. The "Angioedema Quality of Life Questionnaire" has also been shown to be an adequate and effective set of questions for describing HRQoL in a various population of adult patients with HAE [7]. In contrast to the adult population, there is a paucity of data on the HRQoL of children with HAE and their parents' perceptions of the problem.…”

Correction to: Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema

“…Following publication of the original article [ 1 ], the authors identified an error in the author name of Joan Mendivil.…”