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

Bonner N, Abetz-Webb L, Renault L, Caballero T, Longhurst H, Maurer M, Christiansen S, Zuraw B, Icatibant Outcome Survey (IOS) International Executive Committee and the Hereditary Angioedema Association (HAEA) Medical Advisory Boa - Health Qual Life Outcomes (2015)

Bottom Line: 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).The HAE PRO was revised and refined following input from patients and clinical experts.HAE patients across cultures consider the HAE PRO a relevant and appropriate assessment of HAE attacks and treatment.

View Article: PubMed Central - PubMed

Affiliation: Adelphi Values, Adelphi Mill, Bollington, Cheshire, UK. Nicola.Bonner@adelphivalues.com.

ABSTRACT

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.

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Related in: MedlinePlus

HAE PRO conceptual framework
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Fig3: HAE PRO conceptual framework

Mentions: Evidence from the second round of cognitive debriefing interviews was considered alongside patient input from the previous phases of the research and a final instrument was created. Figure 3 presents the conceptual framework for the final instrument with patient quotes confirming the relevance of the HAE PRO. The concepts in the model are color coded to demonstrate the evolution of the instrument over time.Fig. 3


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

Bonner N, Abetz-Webb L, Renault L, Caballero T, Longhurst H, Maurer M, Christiansen S, Zuraw B, Icatibant Outcome Survey (IOS) International Executive Committee and the Hereditary Angioedema Association (HAEA) Medical Advisory Boa - Health Qual Life Outcomes (2015)

HAE PRO conceptual framework
© Copyright Policy - open-access
Related In: Results  -  Collection

License 1 - License 2
Show All Figures
getmorefigures.php?uid=PMC4487558&req=5

Fig3: HAE PRO conceptual framework
Mentions: Evidence from the second round of cognitive debriefing interviews was considered alongside patient input from the previous phases of the research and a final instrument was created. Figure 3 presents the conceptual framework for the final instrument with patient quotes confirming the relevance of the HAE PRO. The concepts in the model are color coded to demonstrate the evolution of the instrument over time.Fig. 3

Bottom Line: 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).The HAE PRO was revised and refined following input from patients and clinical experts.HAE patients across cultures consider the HAE PRO a relevant and appropriate assessment of HAE attacks and treatment.

View Article: PubMed Central - PubMed

Affiliation: Adelphi Values, Adelphi Mill, Bollington, Cheshire, UK. Nicola.Bonner@adelphivalues.com.

ABSTRACT

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.

Show MeSH
Related in: MedlinePlus