Publications, Books, Book Chapters and Reviews by Prof. Marcus Maurer, MD

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

Filename 217. Netchiporouk et al., Man. pediatr. CS and phys. urt. Omalizumab, PAI 2015.pdf
Filesize 285 KB
Version o.217
Date added July 30, 2020
Downloaded 0 times
Category Original Work
Tags Cognitive debriefing, Concept elicitation, Content validity, Hereditary Angioedema, Patient, Patient reported outcome, Qualitative, Questionnaire
Authors Netchiporouk, E., Nguyen, C. H., Thuraisingham, T., Jafarian, F., Maurer, M. and Ben-Shoshan, M.
Citation Netchiporouk, E., Nguyen, C. H., Thuraisingham, T., Jafarian, F., Maurer, M. and Ben-Shoshan, M.: Management of pediatric chronic spontaneous and physical urticaria patients with Omalizumab: case series. Pediatr. Allergy Immunol. 2015: 26; 585-588.
Corresponding authors Ben-Shoshan, M.
DocNum O.217
DocType PDF
Edition; Page 26; 585-588
IF 3.94
Publisher Pediatr. Allergy Immunol.
ReleaseDate 2015

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.

(Last update: 02.2021)

Number of publications (original work and reviews) in peer-reviewed journals: 601
Number of original publications in peer-reviewed journals: 432
Number of reviews in peer-reviewed journals: 169
Cumulative IF of publications (original work & reviews) in peer-reviewed journals: 3326,21
Cumulative IF for original publications in peer-reviewed journals: 2659,47
Cumulative IF for reviews in peer-reviewed journals: 666,74
Citations, Hirsch index: (view on Web of Science) 24104