title: Treatment of hereditary angioedema with nanofiltered C1-esterase inhibitor concentrate (Cetor®): multi-center phase II and III studies to assess pharmacokinetics, clinical efficacy and safety.
abstract: From 1997, plasma-derived C1-inhibitor concentrate (Cetor®) has been available to HAE and AAE patients. Recently, a virus reducing 15 nm nanofiltration step has been introduced in the production process. A randomized, double-blind controlled cross-over study was performed to compare the pharmacokinetics (PK) of nanofiltered (C1-INH-NF) with conventional C1-inhibitor (C1-INH). Efficacy and safety were investigated in an open-label, on-demand and a prophylactic study. No differences in pharmacokinetic parameters between C1-INH and C1-INH-NF were found (13 non-symptomatic HAE patients). Both C1-inhibitor products equally increased plasma C4 levels. In the on-demand study, 14 acute angioedema attacks in 8 patients were analyzed. In the prophylactic study, 1 AAE and 5 HAE patients experienced in total 31 attacks during 748 observation days. In total 180,000 units of C1-INH-NF were administered. No product-related adverse events occurred, and no anti-C1-antibodies were induced. Nanofiltration in the production process of C1-inhibitor did not affect the pharmacokinetics, efficacy, and safety.
authors.affiliation: Department of Internal Medicine, Academic Medical Centre, Amsterdam, The Netherlands.
authors.forename: J J
authors.initials: JJ
authors.lastname: Hofstra
authors.forename: I
authors.initials: I
authors.lastname: Kleine Budde
authors.forename: E
authors.initials: E
authors.lastname: van Twuyver
authors.forename: G
authors.initials: G
authors.lastname: Choi
authors.forename: M
authors.initials: M
authors.lastname: Levi
authors.forename: F W G
authors.initials: FW
authors.lastname: Leebeek
authors.forename: J G R
authors.initials: JG
authors.lastname: de Monchy
authors.forename: P F
authors.initials: PF
authors.lastname: Ypma
authors.forename: R J
authors.initials: RJ
authors.lastname: Keizer
authors.forename: A D R
authors.initials: AD
authors.lastname: Huitema
authors.forename: P F W
authors.initials: PF
authors.lastname: Strengers
mesh_terms: D000328:Adult; D054179:Angioedemas, Hereditary / Q000188:drug therapy* / Q000517:prevention & control; D050718:Complement C1 Inhibitor Protein / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged
chemical_list: D050718:Complement C1 Inhibitor Protein