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

Journal of Patient-Reported Outcomes
Magdalena Vanya1, Maureen Watt2, Saeid Shahraz1, Charlotte E Kosmas3, Stephanie Rhoten4, Sara Costa-Cabral5, Joan Mendívil2, Giovanna Devercelli6, Karsten Weller7
1ICON plc, South San Francisco, CA, USA
2Takeda Pharmaceuticals International AG, Zurich, Switzerland
3ICON plc, Reading, UK
4IQVIA, San Francisco, CA, USA
5Mapi Research Trust, Lyon, France
6Takeda Development Center Americas, Inc., Lexington, MA, USA
7Institute of Allergology, Charité – Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany

Tóm tắt

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.

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Journal of Patient-Reported Outcomes

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