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

Journal of Patient-Reported Outcomes

Research

Charlotte E. Kosmas¹  Stephanie Rhoten²  Magdalena Vanya³  Saeid Shahraz⁴  Karsten Weller⁵  Sara Costa-Cabral⁶  Giovanna Devercelli⁷  Maureen Watt⁸  Joan Mendivil⁸ 

[1] ICON plc, Reading, UK;ICON plc, South San Francisco, CA, USA;IQVIA, San Francisco, CA, USA;ICON plc, South San Francisco, CA, USA;Takeda Development Center Americas, Inc, Lexington, MA, USA;ICON plc, South San Francisco, CA, USA;Tufts Medical Center Institute for Clinical Research and Health Policy, Boston, MA, USA;Institute of Allergology, Charité – Universitätsmedizin Berlin, corporate member of Freie, Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany;Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Immunology and Allergology, Berlin, Germany;Mapi Research Trust, Lyon, France;Takeda Development Center Americas, Inc, Lexington, MA, USA;Takeda Pharmaceuticals International AG, Zurich, Switzerland;

关键词: Content validity;    Hereditary angioedema;    Patient-reported outcomes;    Validity;    Psychometric;    Quality of life;   

DOI  :  10.1186/s41687-023-00576-w

 received in 2022-11-08, accepted in 2023-03-14,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundThere 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.MethodsTo 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.ResultsInterviews 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.ConclusionsQualitative 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.

【 授权许可】

CC BY   
© The Author(s) 2023. corrected publication 2023

【 图 表 】

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