Cost-Consequences Analysis of the Long-Term Prophylaxis In A Type 1 Von Willebrand Disease Patient With Recurrent Bleedings In Italy.

Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, caused by a deficiency or abnormality of the Von Willebrand Factor (VWF). The mainstays of treatment are therapy with Desmopressin (DDAVP) and replacement therapy with Factor VIII-containing VWF product (FVIII/VWF). Therapy with FVIII/VWF can be administered as Long-term prophylaxis (LTP) in the more severe forms of the disease in order to control recurrent bleeding and to prevent life-threatening hemorrhages. However, FVIII/VWF is not enough effective in some cases. Furthermore repeated infusions of FVIII/VWF may increase the risk of thromboembolic events. The objective of the analysis is to assess the economic impact of VWF concentrate almost devoid of FVIII as an alternative to FVIII/VWF for a patient on LTP with recurrent bleedings. Objectives