title: C1-inhibitor therapy for hereditary angioedema attacks: prospective patient assessments of health-related quality of life.
abstract: C1-inhibitor (INH) concentrate, which is recommended as first-line treatment for acute hereditary angioedema (HAE) attacks in many countries, was recently approved in the United States. We sought to solicit patients' feedback about their health-related quality of life (HRQoL) while  being treated with C1-INH concentrate for acute HAE attacks under real-world conditions, as well as the personal impact of the availability of C1-INH on lifestyle and mental health domains. Subjects enrolled in an open-label study of C1-INH at 20 U/kg for acute HAE attacks were invited to  participate in a prospectively designed survey to solicit "real-time" patient responses that were collected via an interactive voice response service or online with a personal computer. Eighteen subjects submitted 60 quarterly HRQoL and treatment impact survey responses  over 29 months. Seventeen of 18 patients responding reported mean short form 12 HRQoL scores that were within a normal range. More than one-half indicated that C1-INH availability made them feel somewhat or much better, and >80% reported having a better outlook on the future and feeling  more secure about the danger of life-threatening attacks. These data confirm a high level of HRQoL and a positive impact in lifestyle and emotional domains among patients who were treated for acute attacks of HAE with C1-INH concentrate.
authors.affiliation: Division of Allergy and Immunology, Creighton University School of Medicine, Omaha, NE 68131, USA. AGAINDRABEWTRA@creighton.edu
authors.forename: Againdra K
authors.initials: AK
authors.lastname: Bewtra
authors.forename: Robyn J
authors.initials: RJ
authors.lastname: Levy
authors.forename: Kraig W
authors.initials: KW
authors.lastname: Jacobson
authors.forename: Richard L
authors.initials: RL
authors.lastname: Wasserman
authors.forename: Thomas
authors.initials: T
authors.lastname: Machnig
authors.forename: Timothy J
authors.initials: TJ
authors.lastname: Craig
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D054179:Angioedemas, Hereditary / Q000188:drug therapy*; D002648:Child; D050718:Complement C1 Inhibitor Protein / Q000008:administration & dosage* / Q000627:therapeutic use; D005260:Female; D006801:Humans; D008297:Male; D008603:Mental Health; D008875:Middle Aged; D011446:Prospective Studies; D011788:Quality of Life; D011795:Surveys and Questionnaires; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D050718:Complement C1 Inhibitor Protein