[Cost-minimization analysis of replacement therapy in the treatment of von Willebrand disease]

BACKGROUND: Replacement therapy with von Willebrand factor (VWF)/factor VIII (FVIII) concentrates represents an effective approach for patients with von Willebrand disease (VWD) who are unresponsive to desmopressin. However, various concentrates are available, with heterogeneous VWF content and VWF/FVIII ratio.AIM: To compare the costs associated to the replacement therapy with VWF/FVIII concentrates in Italy.METHODS: A cost-minimization analysis was performed to compare the pharmaceutical costs per patient of alternatives available for replacement therapy of VWD in the prospective of the Italian National Health Service. For each alternative the analysis calculated the number of vials, and relative costs, required to reach the target levels of VWF:RCo in patients who undergone to major surgery, minor surgery, spontaneous bleeding and prophylaxis.RESULTS: Haemate P® is associated with the lowest FVIII dosage, numbers of vials used and costs in all the clinical situations and at all the dosages considered. With Haemate P® the average costs in major surgery, minor surgery, spontaneous bleeding, and prophylaxis was € 710.94, € 592.45, € 473.96, and € 592.45, respectively. While the costs associated to Fanhdi®, Wilate®, and Wilfactin® was: € 1,309.28, € 1,071.23, € 952.20, and € 1,190.25; € 1,512.45, € 1,344.40, € 1,176.35, and € 1,344.40; € 3,814.09, € 3,269.22, € 3,269.22, and € 3,814.09.CONCLUSIONS: Treatment with Haemate P®, which presents a low FVIII content, allows to reach the target level of VWF:RCo with a lower number of vials and lower costs for the NHS.[Article in Italian]