The treatment with this formulation was proved effective overall

The treatment with this formulation was proved effective overall in the three treatment modalities considered, and the majority of patients including patients with type 3 VWD achieved haemostatic responses rated as ‘good’ to ‘excellent’, with no significant differences between disease subtypes. These results confirmed those of a preplanned ad interim analysis of the study

[12]. The new formulation was therefore at least as effective as the previous one, also associated with clinical responses rated excellent/good in the majority of patients [9, 13-17]. Our efficacy findings were also in line with those of recently published studies with other commercially available VWF/FVIII MK-8669 manufacturer concentrates [18-20]. During the 24-month follow-up period, there was an increase in the number of patients receiving Haemate® P VR for long-term prophylaxis (n = 31) compared with those

on prophylaxis at baseline (n = 16). The role of prophylaxis with VWF/FVIII concentrates in VWD is still a matter of debate. So far, few studies Buparlisib clinical trial have evaluated the use of secondary prophylaxis in VWD. [21-27]. In the study by Berntorp et al. [23] in 35 patients with VWD, mostly with type 3 VWD, prophylaxis was associated with a substantial decrease in the annual number of bleeding events [23, 24]. Also, patients who started prophylaxis at a young age had no joint bleeds and no clinical signs of arthropathy. Similar results were obtained in a recently published cohort study in 32 patients with a median prophylaxis duration of 3 years leading to the resolution of recurrent bleeding in 31 of the 32 patients [21]. Our results show that prophylactic treatment with Haemate® P VR, both for short-term and long-term bleeding prevention, was effective across all disease subtypes. To our knowledge, the treatment of VWD has not been evaluated in pharmacoeconomic analysis

so far. This type of analysis is strongly needed and is likely to contribute also to establishing the role of long-term prophylaxis in the treatment of VWD. We thus collected data describing the impact this website of VWD on a set of pharmacoeconomic variables. During the 24-month follow-up almost half of the patients lost days of school or work and about one-third was hospitalized because of the disease, whereas another third underwent invasive procedures made necessary by the condition. As no previous studies have addressed this aspect it is difficult to make any comparison. However, these figures are in keeping with the presence of significant bleeding histories and morbidity, as shown by the high average BS observed at enrolment. Haemate® P VR was well-tolerated and the switch to this new formulation was not associated with any serious or unexpected adverse event, including thromboembolic events or inhibitor development, in agreement with previous findings [12].

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