title: Efficacy and safety of an intravenous C1-inhibitor concentrate for long-term prophylaxis in hereditary angioedema.
abstract: BACKGROUND
  The plasma-derived, pasteurized, nanofiltered C1-inhibitor concentrate (pnfC1-INH) is approved in the United States as an intravenous (IV) on-demand treatment for hereditary angioedema (HAE) attacks, and, in Europe, as on demand and short-term prophylaxis.
  
  
  OBJECTIVE
  This analysis evaluated Berinert Patient Registry data regarding IV pnfC1-INH used as long-term prophylaxis (LTP).
  
  
  METHODS
  The international registry (2010-2014) collected prospective and retrospective usage, dosing, and safety data on individuals who used pnfC1-INH for any reason.
  
  
  RESULTS
  The registry included data on 47 subjects (80.9% female subjects; mean age, 44.8 years), which reflected 4082 infusions categorized as LTP and a total of 430.2 months of LTP administration. The median absolute dose of pnfC1-INH given for LTP was 1000 IU (range, 500-3000 IU), with a median time interval between infusion and a subsequent pnfC1-INH-treated attack of 72.0 hours (range, 0.0-166.4 hours). Fifteen subjects (31.9%) had no pnfC1-INH-treated HAE attacks within 7 days after pnfC1-INH infusion for LTP; 32 subjects (68.1%) experienced 246 attacks, with rates of 0.06 attacks per infusion and 0.57 attacks per month. A total of 81 adverse events were reported in 16 subjects (34.0%) (0.02 events per infusion; 0.19 events per month); only 3 adverse events were considered related to pnfC1-INH (noncardiac chest pain, postinfusion headache, deep vein thrombosis in a subject with an IV port).
  
  
  CONCLUSIONS
  In this international registry, IV pnf-C1-INH given as LTP for HAE was safe and efficacious, with a low rate of attacks that required pnfC1-INH treatment, particularly within the first several days after LTP administration.
authors.forename: Timothy
authors.initials: T
authors.lastname: Craig
authors.forename: Ralph
authors.initials: R
authors.lastname: Shapiro
authors.forename: Arthur
authors.initials: A
authors.lastname: Vegh
authors.forename: James W
authors.initials: JW
authors.lastname: Baker
authors.forename: Jonathan A
authors.initials: JA
authors.lastname: Bernstein
authors.forename: Paula
authors.initials: P
authors.lastname: Busse
authors.forename: Markus
authors.initials: M
authors.lastname: Magerl
authors.forename: Inmaculada
authors.initials: I
authors.lastname: Martinez-Saguer
authors.forename: Marc A
authors.initials: MA
authors.lastname: Riedl
authors.forename: William
authors.initials: W
authors.lastname: Lumry
authors.forename: Debora
authors.initials: D
authors.lastname: Williams-Herman
authors.forename: Jonathan
authors.initials: J
authors.lastname: Edelman
authors.forename: Henrike
authors.initials: H
authors.lastname: Feuersenger
authors.forename: Thomas
authors.initials: T
authors.lastname: Machnig
authors.forename: Mikhail
authors.initials: M
authors.lastname: Rojavin