Treatment of hereditary angioedema with nanofiltered C1-esterase inhibitor concentrate (Cetor (R)): Multi-center phase II and III studies to assess pharmacokinetics, clinical efficacy and safety

JJ Hofstra, IK Budde, E van Twuyver, G Choi, M Levi, Frank Leebeek, JGR de Monchy, PF Ypma, RJ Keizer, ADR Huitema, PFW Strengers

Research output: Contribution to journalArticleAcademicpeer-review

19 Citations (Scopus)

Abstract

From 1997, plasma-derived C1-inhibitor concentrate (Cetor (R)) 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 ME and 5 RAE 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. (C) 2011 Elsevier Inc. All rights reserved.
Original languageUndefined/Unknown
Pages (from-to)280-290
Number of pages11
JournalClinical Immunology
Volume142
Issue number3
DOIs
Publication statusPublished - 2012

Cite this