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

J. J. Hofstra, I. Kleine Budde*, E. van Twuyver, G. Choi, M. Levi, F. W. G. Leebeek, J. G. R. de Monchy, P. F. Ypma, R. J. Keizer, A. D. R. Huitema, P. F. W. Strengers

*Corresponding author for this work

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    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 languageEnglish
    Pages (from-to)280-290
    Number of pages11
    JournalClinical Immunology
    Volume142
    Issue number3
    DOIs
    Publication statusPublished - Mar-2012

    Keywords

    • Plasma-derived C1-inhibitor
    • Angioedema
    • Nanofiltration
    • INTERNATIONAL CONSENSUS ALGORITHM
    • UPPER AIRWAY-OBSTRUCTION
    • C1-INHIBITOR CONCENTRATE
    • ANGIONEUROTIC-EDEMA
    • REPLACEMENT THERAPY
    • TERM PROPHYLAXIS
    • ACID THERAPY
    • HEPATITIS-C
    • DEFICIENCY
    • ATTACKS

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