dc.contributor.author
Maurer, Marcus
dc.contributor.author
Caballero, T.
dc.contributor.author
Aberer, W.
dc.contributor.author
Zanichelli, A.
dc.contributor.author
Bouillet, L.
dc.contributor.author
Bygum, A.
dc.contributor.author
Grumach, A.S.
dc.contributor.author
Botha, J.
dc.contributor.author
Andresen, I.
dc.contributor.author
Longhurst, H.J.
dc.contributor.author
IOS Study Group
dc.date.accessioned
2022-12-01T13:53:09Z
dc.date.available
2022-12-01T13:53:09Z
dc.identifier.uri
https://refubium.fu-berlin.de/handle/fub188/37124
dc.identifier.uri
http://dx.doi.org/10.17169/refubium-36837
dc.description.abstract
Background: Hereditary angioedema due to C1 inhibitor deficiency (HAE-1/2) is a chronic and debilitating disease. The unpredictable clinical course represents a significant patient burden.
Objective: To analyse longitudinal registry data from the Icatibant Outcome Survey (IOS) in order to characterize temporal changes in disease activity in patients with HAE-1/2.
Methods: Icatibant Outcome Survey (NCT01034969) is an international observational registry monitoring the clinical outcomes of patients eligible for icatibant treatment. The current analyses are based on data collected between July 2009 and July 2019. Retrospective data for attacks recorded in the 12 months prior to IOS enrolment and for each 12-month period up to 7 years were analysed.
Results: Included patients reported angioedema attacks without long-term prophylaxis (LTP; n = 315) and with LTP (n = 292) use at the time of attack onset. Androgens were the most frequently used LTP option (80.8%). At the population level, regardless of LTP use, most patients (52-80%) reporting <5 attacks in Year 1 continued experiencing this rate; similarly, many patients (25-76%) who reported high attack frequency continued reporting ≥10 attacks/year. However, year on year, 31-51% of patients experienced notable changes (increase/decrease of ≥5 attacks) in annual attack frequency. Of patients who reported an absolute change of ≥10 attacks from Year 1 to 2, 17-50% continued to experience a change of this magnitude in subsequent years.
Conclusion: At the population level, attack frequency was generally consistent over 7 years. At the small group level, 28.8-34.5% of patients reported a change in attack frequency of ≥5 attacks from Year 1 to Year 2; up to half of these patients continued to experience this magnitude of variation in disease activity in later years, reflecting high intra-patient variability.
en
dc.rights.uri
https://creativecommons.org/licenses/by-nc/4.0/
dc.subject
hereditary angioedema type 1/2
en
dc.subject
Icatibant Outcome Survey
en
dc.subject.ddc
600 Technik, Medizin, angewandte Wissenschaften::610 Medizin und Gesundheit::610 Medizin und Gesundheit
dc.title
Variability of disease activity in patients with hereditary angioedema type 1/2: longitudinal data from the Icatibant Outcome Survey
dc.type
Wissenschaftlicher Artikel
dcterms.bibliographicCitation.doi
10.1111/jdv.17654
dcterms.bibliographicCitation.journaltitle
Journal of the European Academy of Dermatology and Venereology
dcterms.bibliographicCitation.number
12
dcterms.bibliographicCitation.originalpublishername
Wiley
dcterms.bibliographicCitation.pagestart
2421
dcterms.bibliographicCitation.pageend
2430
dcterms.bibliographicCitation.volume
35
refubium.affiliation
Charité - Universitätsmedizin Berlin
refubium.funding
DEAL Wiley
refubium.resourceType.isindependentpub
no
dcterms.accessRights.openaire
open access
dcterms.bibliographicCitation.pmid
34506666
dcterms.isPartOf.issn
0926-9959
dcterms.isPartOf.eissn
1468-3083