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.

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 / G. Castaman, A. Coppola, E. Zanon, E. Boeri, M. Musso, S. Siragusa, A.B. Federici, G. Mancuso, G. Barillari, C. Biasoli, G. Feola, M. Franchini, S. Moratelli, G. Gamba, P. Schinco, L. Valdrè, A. Dragani, G. Mazzucconi, A. Tagliaferri, M. Morfini. - In: HAEMOPHILIA. - ISSN 1351-8216. - 19:1(2013 Jan), pp. 82-88. [10.1111/hae.12005]

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

A.B. Federici;
2013

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.
Formulation switch; Haemate P; Observational study; Pasteurized VWF/FVIII concentrate; Volume-reduced infusion; Von Willebrand disease
Settore MED/15 - Malattie del Sangue
gen-2013
Article (author)
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/214750
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