title: Home therapy with intravenous human C1-inhibitor in children and adolescents with hereditary angioedema.
abstract: BACKGROUND
  C1-esterase inhibitor (C1-INH) replacement therapy is the treatment of choice for acute edema attacks in patients with hereditary angioedema (HAE).
  
  
  METHODS
  Our retrospective, observational study assessed the efficacy and safety of home therapy with a human plasma-derived C1-INH concentrate (pC1-INH) in 20 pediatric patients with HAE who had previously been treated with physician-based therapy. While on home therapy, 15 patients received on-demand treatment and five received individual replacement treatment (IRT).
  
  
  RESULTS
  The switch to home therapy did not involve a significant increase in the dose of pC1-INH administered, but there was a significant increase in dosing frequency. Although only two patients were affected, the frequency of laryngeal attacks appeared to decrease on home therapy. All attacks, including laryngeal edema, were treated successfully during home therapy with pC1-INH. The mean annual number of days hospitalized was reduced from 3.8 during physician-based therapy to 0.11 during home therapy. No side effects or injection site complications were reported. The median time from onset of attack to administration of pC1-INH was reduced from 67.5 minutes during physician-based therapy to 15 minutes after switching to home therapy. The corresponding median time to initial symptom relief for all types of attack was reduced from 60 to 40 minutes.
  
  
  CONCLUSIONS
  As in adults, home therapy with pC1-INH is effective and safe in the treatment of HAE attacks in pediatric patients; a larger, randomized study should ideally confirm our findings before this approach can be considered the standard of care for pediatric patients.
authors.affiliation: Johann Wolfgang Goethe University Hospital, Frankfurt am Main, Germany.
authors.forename: Wolfhart
authors.initials: W
authors.lastname: Kreuz
authors.forename: Eva
authors.initials: E
authors.lastname: Rusicke
authors.forename: Inmaculada
authors.initials: I
authors.lastname: Martinez-Saguer
authors.forename: Emel
authors.initials: E
authors.lastname: Aygören-Pürsün
authors.forename: Christine
authors.initials: C
authors.lastname: Heller
authors.forename: Thomas
authors.initials: T
authors.lastname: Klingebiel
mesh_terms: D000293:Adolescent; D054179:Angioedemas, Hereditary / Q000188:drug therapy*; D002648:Child; D003174:Complement C1 Inactivator Proteins / Q000008:administration & dosage* / Q000627:therapeutic use*; D050718:Complement C1 Inhibitor Protein; D005260:Female; D006699:Home Care Services; D006801:Humans; D007275:Injections, Intravenous; D008297:Male; D012189:Retrospective Studies
chemical_list: D003174:Complement C1 Inactivator Proteins; D050718:Complement C1 Inhibitor Protein; C469952:SERPING1 protein, human