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- Title
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.
- 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.
- Abstract
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® 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 Haemate ® P VR 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® P was easy to perform and infusion duration was decreased twofold compared with the previous formulation. Volume-reduced Haemate® P was at least as effective and well-tolerated as the previous formulation.
- Subjects
VON Willebrand disease treatment; VON Willebrand factor; BLOOD coagulation factors; HEMORRHAGE; PHARMACOKINETICS
- Publication
Haemophilia, 2013, Vol 19, Issue 1, p82
- ISSN
1351-8216
- Publication type
Article
- DOI
10.1111/hae.12005