Efficacy and safety during formulation switch of a pasteurized VWF/FVIII concentrate: results from an Italian prospective observational study in patients with von Willebrand disease. (7th September 2012)

• Record Type: Journal Article
• Authors: Castaman, G.; Coppola, A.; Zanon, E.; Boeri, E.; Musso, M.; Siragusa, S.; Federici, A. B.; Mancuso, G.; Barillari, G.; Biasoli, C.; Feola, G.; Franchini, M.; Moratelli, S.; Gamba, G.; Schinco, P.; Valdrè, L.; Dragani, A.; Mazzucconi, G.; Tagliaferri, A.; Morfini, M.
• Is Part Of: Haemophilia. Volume 19:Number 1(2013:Jan.)
• Page Start: 82
• Page End: 88
• Publication Date: 2012-09-07
• Subjects: Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.12005
• Languages: English
• ISSNs: 1351-8216

Abstract

<abstract abstract-type="main" id="hae12005-abs-0001"> <title>Summary</title> <p>Von Willebrand disease (VWD) is an inherited bleeding disorder caused by the quantitative or qualitative deficiency of von Willebrand factor (VWF). Replacement therapy with plasma‐derived VWF/factor VIII (FVIII) concentrates is required in patients unresponsive to desmopressin. To assess the efficacy, safety and ease of use of a new, volume‐reduced (VR) formulation of VWF/FVIII concentrate Haemate<sup>®</sup> P in patients requiring treatment for bleeding or prophylaxis for recurrent bleeding or for invasive procedures. Pharmacoeconomic variables were also recorded. Data were analysed using descriptive statistics. This was a multicentre, prospective, observational study. Consecutively enrolled patients received <underline>Haemate</underline><sup><underline>®</underline></sup><underline>P</underline><underline>VR</underline> according to their needs, and were followed for 24 months. Of the 121 patients enrolled, 25.6% had type 3 VWD and more than 40% had severe disease. All patients were followed for 2 years, for a total of 521 visits. On‐demand treatment was given to 61.9% of patients, secondary long‐term prophylaxis to 25.6% and prophylaxis for surgery, dental or invasive procedures to 45.5%. The response to treatment was rated as good to excellent in >93–99% of interventions. The new formulation was well tolerated by all patients with no report of drug‐related adverse events. The switch to volume‐reduced Haemate<sup>®</sup> P was easy to perform and infusion duration was decreased twofold compared with the previous formulation. Volume‐reduced Haemate<sup>®</sup> P was at least as effective and well‐tolerated as the previous formulation.</p> </abstract>