title: Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks.
abstract: BACKGROUND
  Hereditary angioedema caused by C1 esterase inhibitor deficiency is a rare disorder.
  
  
  OBJECTIVE
  To compare the efficacy of pasteurized C1 esterase inhibitor concentrate (Berinert, CSL Behring) at intravenous doses of 10 or 20 U/kg body weight with placebo in the treatment of single, acute abdominal or facial attacks in patients with hereditary angioedema.
  
  
  METHODS
  This was a randomized, double-blind, placebo-controlled study in 125 patients with type I or II hereditary angioedema. The primary outcome was time from start of treatment to onset of symptom relief. Secondary outcomes were time to complete resolution, proportion of patients with worsened intensity of angioedema symptoms between 2 and 4hours after treatment, and number of vomiting episodes within 4 hours.
  
  
  RESULTS
  Median time to onset of relief was significantly shorter with C1 esterase inhibitor concentrate at a dose of 20 U/kg than with placebo (0.5 vs 1.5 hours; P = .0025), whereas with 10 U/kg, the time to onset of relief was only slightly shorter than with placebo (1.2 vs 1.5 hours; P = .2731). Compared with placebo, the reduction in time to onset of relief was greatest for severe attacks (0.5 vs 13.5 hours). The secondary outcomes consistently supported the efficacy of the 20 U/kg dose. C1 esterase inhibitor concentrate was safe and well tolerated. No seroconversions were observed for HIV, hepatitis virus, or human B19 virus.
  
  
  CONCLUSIONS
  C1 esterase inhibitor concentrate given intravenously at a dose of 20 U/kg is an effective and safe treatment for acute abdominal and facial attacks in patients with hereditary angioedema, with a rapid onset of relief.
authors.affiliation: Penn State University College of Medicine, Hershey, Pa., USA.
authors.forename: Timothy J
authors.initials: TJ
authors.lastname: Craig
authors.forename: Robyn J
authors.initials: RJ
authors.lastname: Levy
authors.forename: Richard L
authors.initials: RL
authors.lastname: Wasserman
authors.forename: Againdra K
authors.initials: AK
authors.lastname: Bewtra
authors.forename: David
authors.initials: D
authors.lastname: Hurewitz
authors.forename: Krystyna
authors.initials: K
authors.lastname: Obtułowicz
authors.forename: Avner
authors.initials: A
authors.lastname: Reshef
authors.forename: Bruce
authors.initials: B
authors.lastname: Ritchie
authors.forename: Dumitru
authors.initials: D
authors.lastname: Moldovan
authors.forename: Todor
authors.initials: T
authors.lastname: Shirov
authors.forename: Vesna
authors.initials: V
authors.lastname: Grivcheva-Panovska
authors.forename: Peter C
authors.initials: PC
authors.lastname: Kiessling
authors.forename: Heinz-Otto
authors.initials: HO
authors.lastname: Keinecke
authors.forename: Jonathan A
authors.initials: JA
authors.lastname: Bernstein
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000368:Aged; D054179:Angioedemas, Hereditary / Q000188:drug therapy*; D002648:Child; D050718:Complement C1 Inhibitor Protein / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D051056:Complement Inactivating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D007275:Injections, Intravenous; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D055815:Young Adult
chemical_list: D050718:Complement C1 Inhibitor Protein; D051056:Complement Inactivating Agents