Development and content validity testing of a patient-reported outcomes questionnaire for the assessment of hereditary angioedema in observational studies

Health and Quality of Life Outcomes

Bruce Zuraw¹  Sandra Christiansen⁷  Marcus Maurer²  Hilary Longhurst⁵  Teresa Caballero⁴  Lydie Renault⁶  Linda Abetz-Webb³  Nicola Bonner³ 

[1] San Diego Veterans Hospital, San Diego, CA, USA;Department of Dermatology and Allergy, Allergie-Centrum-Charité, Charité - Universitätsmedizin Berlin, Berlin, Germany;Adelphi Values, Adelphi Mill, Bollington, Cheshire, UK;University Hospital, La Paz, Hospital La Paz Institute for Health Research (IdiPaz), Biomedical Research Network on Rare Diseases-U754 (CIBERER), Madrid, Spain;Department of Immunology, Barts Health NHS Trust, London, UK;Shire International GmbH, Zug, Switzerland;Medicine Division Allergy/ Immunology and US HAEA Angioedema Center, University of California, San Diego, CA, USA

关键词: Content validity;    Cognitive debriefing;    Concept elicitation;    Patient;    Qualitative;    Questionnaire;    Patient reported outcome;    Hereditary angioedema;   

Others  :  1218512
DOI  :  10.1186/s12955-015-0292-7

 received in 2014-11-25, accepted in 2015-06-22,  发布年份 2015

【 摘 要 】

Background

Hereditary Angioedema (HAE), a rare genetic disease, manifests as intermittent, painful attacks of angioedema. Attacks vary in frequency and severity and include skin, abdominal and life-threatening laryngeal swellings. This study aimed to develop a patient reported outcome (PRO) tool for the assessment of HAE attacks, including their management and impact on patients’ lives, for use in clinical studies, or by physicians in general practice.

Methods

The results of open-ended face to face concept elicitation interviews with HAE patients in Argentina (n = 10) and the US (n = 33) were used to develop the first draft questionnaire of the HAE patient reported outcomes questionnaire (HAE PRO). Subsequently, in-depth cognitive debriefing interviews were performed with HAE patients in the UK (n = 10), Brazil (n = 10), Germany (n = 11) and France (n = 12). Following input from eight multinational clinical experts further cognitive interviews were conducted in the US (n = 12) and Germany (n = 12). Patients who experienced abdominal, cutaneous or laryngeal attacks of varying severity levels were included in all rounds of interviews. Across the rounds of interviews patients discussed their HAE attack symptoms, impacts and treatments. Cognitive debriefing interviews explored patient understanding and relevance of questionnaire items. All interviews were conducted face to face following a pre-defined semi-structured interview guide in the patient’s native language.

Results

Patients reported a variety of HAE symptoms, attack triggers, warning signs, attack impacts and treatment options which were used to develop the HAE PRO. The HAE PRO was revised and refined following input from patients and clinical experts. The final 18-item HAE PRO provides an assessment of the HAE attack experience including symptoms, impacts, treatment requirements, healthcare resource use and loss of productivity caused by HAE attacks.

Conclusions

Patient and expert input has contributed to the development of a content valid questionnaire that assesses concepts important to HAE patients globally. HAE patients across cultures consider the HAE PRO a relevant and appropriate assessment of HAE attacks and treatment.

【 授权许可】

   
2015 Bonner et al.

【 图 表 】

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