title: A cross-sectional questionnaire assessing patient and physician use of short-term prophylaxis for hereditary angioedema.
abstract: BACKGROUND
  Current guidelines recommend short-term prophylaxis (STP) before invasive procedures to prevent hereditary angioedema (HAE) attacks; however, adherence to these guidelines may be variable because this indication lacks Food and Drug Administration approval in the United States.
  
  
  OBJECTIVE
  To ascertain the STP experiences of patients with HAE and HAE-treating physicians.
  
  
  METHODS
  Online questionnaires focusing on STP experiences were distributed by the US Hereditary Angioedema Association to the first 250 patients with HAE and to registered HAE-treating physicians. SAS 9.3 was used to perform descriptive statistics and to test the difference between patients who underwent procedures and those who did not using Pearson χ(2) test, Fisher exact test, and 2-sample t test.
  
  
  RESULTS
  For the patient survey, 219 respondents met the criteria for HAE type 1 and 2; 37 (17%) underwent 66 invasive procedures, and all reported receiving STP. Eight patients (22%) reported failed STP, but only 3 required on-demand therapy. For STP, anabolic steroids and plasma-derived C1 inhibitor were the most and second-most commonly used, respectively. For the physician survey, 37 physicians reported caring for 433 patients with HAE. Depending on the procedure, 19% to 54% of physicians used STP and 30% to 86% prescribed on-demand therapy; 69% and 78% of physicians prescribed plasma-derived C1 inhibitor as STP for minimally invasive and invasive procedures, respectively. Physicians reported excellent efficacy for the STP treatments used.
  
  
  CONCLUSIONS
  Physicians reported excellent outcomes using primarily newer STP therapies, namely plasma-derived C1 inhibitor, which was discordant to patient-reported outcomes using older STP therapies, namely anabolic steroids. Well-controlled STP studies are needed to clarify use for patients with HAE in the United States.
authors.affiliation: Division of Allergy and Immunology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
authors.forename: Maya K
authors.initials: MK
authors.lastname: Nanda
authors.affiliation: Division of Immunology, University of Cincinnati, Cincinnati, Ohio.
authors.forename: Umesh
authors.initials: U
authors.lastname: Singh
authors.affiliation: United States Hereditary Angioedema Association, Honolulu, Hawaii.
authors.forename: Joyce
authors.initials: J
authors.lastname: Wilmot
authors.affiliation: Division of Immunology, University of Cincinnati, Cincinnati, Ohio. Electronic address: jonathan.bernstein@uc.edu.
authors.forename: Jonathan A
authors.initials: JA
authors.lastname: Bernstein
mesh_terms: D000799:Angioedema / Q000188:drug therapy / Q000517:prevention & control* / Q000601:surgery; D003174:Complement C1 Inactivator Proteins / Q000627:therapeutic use*; D050718:Complement C1 Inhibitor Protein; D003430:Cross-Sectional Studies; D003613:Danazol / Q000627:therapeutic use*; D004965:Estrogen Antagonists / Q000627:therapeutic use; D006801:Humans; D010361:Patients / Q000706:statistics & numerical data; D010820:Physicians / Q000706:statistics & numerical data; D011795:Surveys and Questionnaires
chemical_list: D003174:Complement C1 Inactivator Proteins; D050718:Complement C1 Inhibitor Protein; D004965:Estrogen Antagonists; C469952:SERPING1 protein, human; D003613:Danazol