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.
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.
authors.affiliation: San Bortolo Hospital, Vicenza, Italy. castaman@hemato.ven.it
authors.forename: G
authors.initials: G
authors.lastname: Castaman
authors.forename: A
authors.initials: A
authors.lastname: Coppola
authors.forename: E
authors.initials: E
authors.lastname: Zanon
authors.forename: E
authors.initials: E
authors.lastname: Boeri
authors.forename: M
authors.initials: M
authors.lastname: Musso
authors.forename: S
authors.initials: S
authors.lastname: Siragusa
authors.forename: A B
authors.initials: AB
authors.lastname: Federici
authors.forename: G
authors.initials: G
authors.lastname: Mancuso
authors.forename: G
authors.initials: G
authors.lastname: Barillari
authors.forename: C
authors.initials: C
authors.lastname: Biasoli
authors.forename: G
authors.initials: G
authors.lastname: Feola
authors.forename: M
authors.initials: M
authors.lastname: Franchini
authors.forename: S
authors.initials: S
authors.lastname: Moratelli
authors.forename: G
authors.initials: G
authors.lastname: Gamba
authors.forename: P
authors.initials: P
authors.lastname: Schinco
authors.forename: L
authors.initials: L
authors.lastname: Valdrè
authors.forename: A
authors.initials: A
authors.lastname: Dragani
authors.forename: G
authors.initials: G
authors.lastname: Mazzucconi
authors.forename: A
authors.initials: A
authors.lastname: Tagliaferri
authors.forename: M
authors.initials: M
authors.lastname: Morfini
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000925:Anticoagulants / Q000009:adverse effects / Q000627:therapeutic use*; D016063:Blood Loss, Surgical / Q000517:prevention & control; D002648:Child; D002675:Child, Preschool; D017281:Cost of Illness; D057915:Drug Substitution; D005169:Factor VIII / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006470:Hemorrhage / Q000517:prevention & control; D006760:Hospitalization / Q000706:statistics & numerical data; D006801:Humans; D007558:Italy; D008297:Male; D008875:Middle Aged; D061352:Pasteurization; D011446:Prospective Studies; D055815:Young Adult; D014842:von Willebrand Diseases / Q000188:drug therapy*; D014841:von Willebrand Factor / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D000925:Anticoagulants; D014841:von Willebrand Factor; D005169:Factor VIII