title: Before and after, the impact of available on-demand treatment for HAE.
abstract: Availability of effective treatment for acute attacks is expected to transform the care of hereditary angioedema (HAE) patients. We felt that it would be of interest to test these assumptions by examining the perceptions of HAE patients regarding the impact that these therapies have had on their lives. Patients at a United States HAE Association summit meeting were asked to rate the burden of HAE currently and compare by recall with 2009 when these therapies were not available. Questions covered five domains: psychological/emotional status, ability to carry out daily activities, fear of suffocation, worry about their children inheriting HAE, and medication side effects. Data were analyzed using Wilcoxon signed-rank tests or analysis of variance. Responses were obtained from 134 self-identified HAE subjects: 85 type I, 21 type II, and 28 with normal C1 inhibitor (C1INH). Burden of disease showed significant improvement in all domains except worry about children inheriting HAE. With the introduction of newer therapies, subjects with the most severe burden of illness improved more than those with milder burdens. However, significant burden of illness remained. The availability of the current treatments has substantially improved the quality of life for HAE patients in the United States, similar to a survey of Danish HAE patients regarding the introduction of home treatment. Nevertheless, our study shows that a substantial burden of illness remains for HAE patients.
authors.affiliation: Department of Medicine, University of California San Diego, La Jolla, CA, USA.
authors.forename: Sandra C
authors.initials: SC
authors.lastname: Christiansen
authors.forename: Anette
authors.initials: A
authors.lastname: Bygum
authors.forename: Aleena
authors.initials: A
authors.lastname: Banerji
authors.forename: Paula
authors.initials: P
authors.lastname: Busse
authors.forename: Henry
authors.initials: H
authors.lastname: Li
authors.forename: William
authors.initials: W
authors.lastname: Lumry
authors.forename: Mark
authors.initials: M
authors.lastname: Davis-Lorton
authors.forename: Jonathan A
authors.initials: JA
authors.lastname: Bernstein
authors.forename: Michael M
authors.initials: MM
authors.lastname: Frank
authors.forename: Anthony
authors.initials: A
authors.lastname: Castaldo
authors.forename: Janet F
authors.initials: JF
authors.lastname: Long
authors.forename: Marc
authors.initials: M
authors.lastname: Riedl
authors.forename: Bruce L
authors.initials: BL
authors.lastname: Zuraw
mesh_terms: D000203:Activities of Daily Living / Q000523:psychology; D054179:Angioedemas, Hereditary / Q000150:complications / Q000188:drug therapy* / Q000523:psychology; D002648:Child; D003718:Denmark; D018450:Disease Progression; D064420:Drug-Related Side Effects and Adverse Reactions / Q000209:etiology / Q000517:prevention & control* / Q000523:psychology; D005239:Fear / Q000523:psychology; D006297:Health Services Accessibility; D006801:Humans; D010465:Perception; D011788:Quality of Life; D016896:Treatment Outcome; D014481:United States