Efficacy and safety of Wilate in paediatric VWD patients under 6 years of age – results of a prospective multicentre clinical study including recovery information

Authors

  • U. Nowak-Göttl,

    Corresponding author
    1. Institute of Clinical Chemistry, Thrombosis & Hemostasis Treatment Center, University Hospital Schleswig-Holstein, Kiel, Germany
    2. Department of Pediatrics, University Hospital Münster, Münster, Germany
    • Correspondence: Ulrike Nowak-Göttl, Institute of Clinical Chemistry, Thrombosis & Hemostasis Treatment Center, University Hospital Schleswig-Holstein, Arnold-Heller-Straße 3 (Haus 17), 24105 Kiel, Germany.

      Tel.: +49 (431) 597 3225; fax: +49 (431) 597 4053;

      e-mail: leagottl@uksh.de

    Search for more papers by this author
  • A. Krümpel,

    1. Department of Pediatrics, University Hospital Münster, Münster, Germany
    Search for more papers by this author
  • A. Russo,

    1. University Hospital, Johannes Gutenberg-Universität Mainz, Mainz, Germany
    Search for more papers by this author
  • M. Jansen

    1. Octapharma Produktionsgesellschaft mbH, Vienna, Austria
    Search for more papers by this author

Summary

Treatment with exogenous von Willebrand factor (VWF) is indicated in patients with von Willebrand disease (VWD) in whom treatment with 1-deamino-8-d-arginine vasopressin/desmopressin is contraindicated. Wilate® is a new generation plasma-derived concentrate of native VWF and coagulation factor VIII (FVIII) (in a physiological 1:1 ratio) developed for the treatment of VWD. This is the first study to report safety, efficacy and in vivo recovery (IVR) data from 15 paediatric patients less than 6 years of age who received Wilate® for either prophylaxis, on-demand treatment or for treatment in surgical procedures during a prospective open-label trial (VWD type 1: 5, type 2A: 1, type 2B: 2, type 3: 6, unknown type: 1 patients). Analysis of IVR for VWF and FVIII suggested an appropriate and consistent rise in coagulation activity after Wilate® administration. Overall efficacy was rated as excellent or good for 99.7% [prophylactic infusions] and 100% [bleeding episodes/surgical procedures]. More than 82% of bleeding episodes resolved after 1 day of treatment, and a Wilate® dosage of 20–50 IU kg−1 was sufficient to achieve haemostasis in 97% of bleeding episodes. All surgical procedures were successfully managed with Wilate®. No thromboembolic events were observed during the study, and no patient developed anti-VWF antibodies or FVIII inhibitors. In conclusion, this study confirms both the expected IVR profile in paediatric patients and the excellent efficacy, tolerability and safety profile of Wilate® observed previously in adults. Wilate® showed excellent efficacy in the treatment of bleeding when used prophylactically or on-demand, and in the treatment of surgical procedures.

Ancillary