title: Nanofiltered human C1 inhibitor concentrate (Cinryze®): in hereditary angioedema.
abstract: Intravenous nanofiltered human C1 inhibitor (C1-INH NF) concentrate (Cinryze®) is used as a direct replacement of deficient levels of plasma C1 inhibitor in patients with hereditary angioedema (HAE). In the EU, C1-INH NF concentrate 1000 U is indicated in the treatment, pre-procedural prevention, and routine prevention of angioedema attacks in adults and adolescents with HAE. Intravenous C1-INH NF concentrate 1000 U effectively relieved angioedema attacks in patients with HAE. In a randomized, double-blind trial in pediatric and adult patients, the median time to onset of unequivocal relief from an attack was significantly shorter with C1-INH NF concentrate than with placebo. In an open-label trial, both unequivocal relief and clinical relief were shown in the majority of attacks within 1 and 4 hours of infusion of C1-INH NF concentrate, regardless of the site (i.e. gastrointestinal, cutaneous, laryngeal, or genitourinary) of the defining symptom. When administered prior to a procedure, open-label intravenous C1-INH NF concentrate 1000 U reduced the incidence of angioedema attacks during and after a variety of dental, surgical, or interventional diagnostic procedures in pediatric and adult patients with HAE. Routine preventative treatment with intravenous C1-INH NF concentrate 1000 U every 3 or 4 days reduced the number of angioedema attacks. In a randomized, double-blind, crossover trial in pediatric and adult patients with HAE, the mean normalized number of attacks per 12-week period was significantly lower during routine prevention with C1-INH NF concentrate than with placebo. Routine prevention with C1-INH NF concentrate reduced the median monthly attack rate from baseline in an open-label trial. Intravenous C1-INH NF concentrate was well tolerated in clinical trials in patients with HAE. No cases of viral transmission were reported.
authors.affiliation: Adis, a Wolters Kluwer Business, Auckland, New Zealand. demail@adis.co.nz
authors.forename: Katherine A
authors.initials: KA
authors.lastname: Lyseng-Williamson
mesh_terms: D000328:Adult; D054179:Angioedemas, Hereditary / Q000188:drug therapy* / Q000517:prevention & control; D002648:Child; D003174:Complement C1 Inactivator Proteins / Q000008:administration & dosage / Q000302:isolation & purification* / Q000493:pharmacokinetics* / Q000627:therapeutic use*; D050718:Complement C1 Inhibitor Protein; D004311:Double-Blind Method; D005260:Female; D005374:Filtration / Q000379:methods*; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D036103:Nanotechnology / Q000379:methods
chemical_list: D003174:Complement C1 Inactivator Proteins; D050718:Complement C1 Inhibitor Protein; C469952:SERPING1 protein, human