title: Health-related quality of life with hereditary angioedema following prophylaxis with subcutaneous C1-inhibitor with recombinant hyaluronidase.
abstract: BACKGROUND
  To estimate health-related quality-of-life changes in patients with hereditary angioedema due to C1-inhibitor (C1-INH) deficiency who received subcutaneous C1-INH with recombinant hyaluronidase (rHuPH20) for attack prophylaxis in a randomized, double-blind, dose-ranging, cross-over study.
  
  
  METHODS
  Patients with type I/II hereditary angioedema received 1000 U of C1-INH with 24,000 U of rHuPH20 or 2000 U of C1-INH with 48,000 U of rHuPH20 every 3-4 days for 8 weeks and then crossed over for another 8-week period. The study was terminated early as a precaution related to non-neutralizing antibodies to rHuPH20. The Angioedema Quality of Life questionnaire (AE-QoL) was administered at weeks 1 and 5 of both periods, and at 1 week after the second treatment period. Changes in AE-QoL scores were calculated over both treatment periods and within each treatment period for patients with ≥4 weeks of treatment.
  
  
  RESULTS
  Forty-one patients had evaluable AE-QoL data, and 22 patients completed treatment. At screening, 43% of the patients were receiving intravenous C1-INH. A significant average AE-QoL total score decline (improvement) of -8.1 (95% confidence interval, -13.7 to -2.5) was observed from baseline to the end of the study, and significant AE-QoL score declines were observed in the Functioning, Fear/Shame, and Nutrition domains. Patients on 2000 U reported higher mean AE-QoL score declines in Functioning and Nutrition domains relative to the 1000 U dose. Overall, 43.9% of all the patients, 45.5% of the study completers, and 46.7% of the nonprophylaxis users at baseline on high treatment doses achieved a reduction in the AE-QoL total score of six points.
  
  
  CONCLUSIONS
  Despite early termination and prestudy prophylactic intravenous C1-INH use by 43% of the patients, improved AE-QoL scores were observed after ≤16 weeks of subcutaneous C1-INH-rHuPH20 prophylaxis.
authors.forename: Karsten
authors.initials: K
authors.lastname: Weller
authors.forename: Marcus
authors.initials: M
authors.lastname: Maurer
authors.forename: Moshe
authors.initials: M
authors.lastname: Fridman
authors.forename: Dylan
authors.initials: D
authors.lastname: Supina
authors.forename: Jennifer
authors.initials: J
authors.lastname: Schranz
authors.forename: Markus
authors.initials: M
authors.lastname: Magerl
mesh_terms: D061605:Administration, Intravenous; D000328:Adult; D050718:Complement C1 Inhibitor Protein / Q000627:therapeutic use*; D051056:Complement Inactivating Agents / Q000627:therapeutic use*; D018592:Cross-Over Studies; D004311:Double-Blind Method; D005260:Female; D006304:Health Status; D056829:Hereditary Angioedema Types I and II / Q000517:prevention & control*; D006801:Humans; D006821:Hyaluronoglucosaminidase / Q000627:therapeutic use*; D055104:Infusions, Subcutaneous; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D011994:Recombinant Proteins; D055502:Secondary Prevention; D011795:Surveys and Questionnaires; D016896:Treatment Outcome
chemical_list: D050718:Complement C1 Inhibitor Protein; D051056:Complement Inactivating Agents; D011994:Recombinant Proteins; D006821:Hyaluronoglucosaminidase