title: Safety and efficacy of C1 esterase inhibitor for acute attacks in children with hereditary angioedema.
abstract: BACKGROUND
  Human plasma-derived nanofiltered C1 esterase inhibitor (C1 INH-nf) is used to treat acute angioedema attacks in patients with hereditary angioedema (HAE), but data regarding use in children are sparse.
  
  
  METHODS
  Patients 2 to <12 years of age, body weight ≥10 kg, with a diagnosis of HAE type I or II, were recruited for a multicenter open-label trial. Patients were recruited into 2 weight categories (10-25 kg, >25 kg). Each weight category included 2 dosing levels: C1 INH-nf (500 units [U], 1000 U) and C1 INH-nf (1000 U, 1500 U), respectively. Patients experiencing an angioedema attack were given a single intravenous dose. Primary efficacy end-point was the onset of unequivocal relief of the defining symptom within 4 h following initiation of C1 INH-nf treatment.
  
  
  RESULTS
  Nine children were treated: 3 (10-25 kg) received 500 U; 3 (>25 kg) received 1000 U; and 3 (>25 kg) received 1500 U. The lower weight/higher dose category (10-25 kg, 1000 U) was not successfully enrolled. All patients completed the study. Most angioedema attacks (n = 5) were abdominal. All patients met the primary end-point; median time to unequivocal symptom relief was 0.5 (range: 0.25-2.5) h. Doses of C1 INH-nf ranged from 20.8 to 51.9 U/kg.
  
  
  CONCLUSIONS
  Treatment of a single angioedema attack with C1 INH-nf doses of 500 U (in patients 10-25 kg), 1000 U, and 1500 U (in patients >25 kg) were well tolerated. Doses of C1 INH-nf <1000 U may be appropriate in some pediatric patients.
authors.affiliation: AARA Research Center, Dallas, TX, USA.
authors.forename: William
authors.initials: W
authors.lastname: Lumry
authors.affiliation: Asthma & Allergy Associates, Colorado Springs, CO, USA.
authors.forename: Daniel
authors.initials: D
authors.lastname: Soteres
authors.affiliation: Marycliff Allergy Specialists, Spokane, WA, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Gower
authors.affiliation: Oregon Allergy Associates, Eugene, OR, USA.
authors.forename: Kraig W
authors.initials: KW
authors.lastname: Jacobson
authors.affiliation: Institute for Allergy and Asthma, Chevy Chase, MD, USA.
authors.forename: H Henry
authors.initials: HH
authors.lastname: Li
authors.affiliation: ViroPharma Incorporated (part of the Shire Group of Companies), Exton, PA, USA.
authors.forename: Hongzi
authors.initials: H
authors.lastname: Chen
authors.affiliation: ViroPharma Incorporated (part of the Shire Group of Companies), Exton, PA, USA.
authors.forename: Jennifer
authors.initials: J
authors.lastname: Schranz
mesh_terms: D000208:Acute Disease; D054179:Angioedemas, Hereditary / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D050718:Complement C1 Inhibitor Protein / Q000627:therapeutic use*; D051056:Complement Inactivating Agents / Q000627:therapeutic use*; D018450:Disease Progression; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D008297:Male; D016896:Treatment Outcome
chemical_list: D050718:Complement C1 Inhibitor Protein; D051056:Complement Inactivating Agents
keywords: C1 esterase inhibitor; acute treatment; children; hereditary angioedema; safety