title: Nanofiltered C1-esterase inhibitor for the acute management and prevention of hereditary angioedema attacks due to C1-inhibitor deficiency in children.
abstract: OBJECTIVE
  To evaluate the use of Cinryze (nanofiltered C1-esterase inhibitor [C1 INH-nf]) for the acute management and prevention of hereditary angioedema attacks in the subgroup of children and adolescents who participated in 2 placebo-controlled and 2 open-label extension studies.
  
  
  METHODS
  In the acute-attack treatment studies, the efficacy of 1000 U of C1 INH-nf (with an additional 1000 U given 1 hour later if needed) was assessed based on the time to the start of symptomatic relief and the proportion of patients experiencing relief within 4 hours of therapy. In the prophylaxis studies, C1 INH-nf 1000 U was given twice weekly, and efficacy was based on the frequency of attacks.
  
  
  RESULTS
  Across 4 studies, 46 children received a total of 2237 C1 INH-nf infusions. The median time to the start of unequivocal relief in the acute-attack treatment study (n = 12) was 30 minutes with C1 INH-nf, compared with 2 hours for placebo. In the open-label extension (n = 22), clinical relief began within 4 hours of therapy in 89% of attacks. In the prophylaxis study (n = 4), the number of attacks was reduced by approximately 2-fold with C1 INH-nf compared with placebo. In the prophylaxis open-label extension (n = 23), the median monthly attack rate decreased from 3.0 before treatment to 0.39 with C1 INH-nf use.
  
  
  CONCLUSIONS
  In children, C1 INH-nf was well tolerated, provided relief from symptoms of hereditary angioedema attacks, and reduced the rate of attacks.
authors: Asthma and Allergy Research Associates, Dallas, TX, USA. Lumrymd@aararesearch.com
authors: William
authors: W
authors: Lumry
authors: Michael E
authors: ME
authors: Manning
authors: David S
authors: DS
authors: Hurewitz
authors: Mark
authors: M
authors: Davis-Lorton
authors: David
authors: D
authors: Fitts
authors: Ira N
authors: IN
authors: Kalfus
authors: Marc E
authors: ME
authors: Uknis
mesh_terms: D000293:Adolescent; D054179:Angioedemas, Hereditary / Q000188:drug therapy* / Q000517:prevention & control; D002648:Child; D002675:Child, Preschool; D050718:Complement C1 Inhibitor Protein / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D005374:Filtration; D056829:Hereditary Angioedema Types I and II / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D016896:Treatment Outcome
chemical_list: D050718:Complement C1 Inhibitor Protein