Observational studyHealth and quality of life outcomes2015
Development and content validity testing of a patient-reported outcomes questionnaire for the assessment of hereditary angioedema in observational studies.
Observational study in Health and quality of life outcomes, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 28 citations in OpenAlex.
- Disease-specific health related quality of life patient reported outcome measures in Genodermatoses: a systematic review and critical evaluation.Orphanet journal of rare diseases · 2017Pooled it
- Impact of lanadelumab on health-related quality of life in patients with hereditary angioedema in the HELP study.Allergy · 2021Trial
- Is hereditary angioedema associated with deficits in emotion regulation? A quantitative study in adult patients.Orphanet journal of rare diseases · 2026Article
- Understanding data visualization techniques in qualitative studies used to develop and validate patient-reported outcome measures: a targeted literature review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Review
- Unmet needs in the management of hereditary angioedema from the perspective of Brazilian patients.The World Allergy Organization journal · 2024Article
- Clinical Characteristics and Quality of Life in a Cohort of Polish Pediatric Patients with Hereditary Angioedema.Children (Basel, Switzerland) · 2024Article
- Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema.Journal of patient-reported outcomes · 2023Article
- The symptom experience of hereditary angioedema (HAE) patients beyond HAE attacks: literature review and clinician interviews.Orphanet journal of rare diseases · 2022Review
- New Instrument for the Evaluation of Prodromes and Attacks of Hereditary Angioedema (HAE-EPA).Clinical reviews in allergy & immunology · 2021Article
- The Enigma of Prodromes in Hereditary Angioedema (HAE).Clinical reviews in allergy & immunology · 2021Review
- Patient-reported Outcome Measures for Angioedema: A Literature Review.Acta dermato-venereologica · 2021Review
- Angioedema quality of life questionnaire (AE-QoL) - interpretability and sensitivity to change.Health and quality of life outcomes · 2019Article
- Health-related quality of life and its risk factors in Chinese hereditary angioedema patients.Orphanet journal of rare diseases · 2019Article
- Patient and observer reported outcome measures to evaluate health-related quality of life in inherited metabolic diseases: a scoping review.Orphanet journal of rare diseases · 2018Article
- Patient perspectives on the use of categories of conditions for decision making about genomic carrier screening results.American journal of medical genetics. Part A · 2018Article
- Hereditary angioedema with normal C1 inhibitor in a French cohort: Clinical characteristics and response to treatment with icatibant.Immunity, inflammation and disease · 2017Article
- High attack frequency in patients with angioedema due to C1-inhibitor deficiency is a major determinant in switching to home therapy: a real-life observational study.Orphanet journal of rare diseases · 2016Article
- Diagnosis and screening of patients with hereditary angioedema in primary care.Therapeutics and clinical risk management · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 5 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundHereditary 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.
methodsThe 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.
resultsPatients 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.
conclusionsPatient 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.
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