Schinco Piercarla, Cultrera Dorina, Valeri Federica, Borchiellini Alessandra, Mantuano Michela, Gorla Francesca, Savarese Alessia, Teruzzi Cristina
Hemostasis and Thrombosis Unit, Molinette Hospital of Turin, Milan, Italy.
Department of Hematology, Hemophilia Regional Reference Center, University Hospital of Catania, Milan, Italy.
Clinicoecon Outcomes Res. 2014 Dec 17;7:17-25. doi: 10.2147/CEOR.S71892. eCollection 2015.
Prophylaxis with von Willebrand factor (VWF)/factor VIII (FVIII) concentrates is a potential approach for patients with severe von Willebrand disease (VWD). As far as we are aware, to date there have been no pharmacoeconomic analyses in order to assess the economic impact of treatments for severe VWD. The analysis presented here estimates the cost-benefit ratio of VWF with a low FVIII content when compared with VWF/FVIII concentrates currently used in Italy for long-term prophylaxis in patients with severe VWD.
A cost-consequence analysis was undertaken to assess the economic impact of the treatment of severe VWD from the perspective both of the Italian National Health Service and society. The analysis was based on four case reports of long-term prophylaxis with VWD with VWF/FVIII concentrates and VWF with a low FVIII content. The costs per patient included direct and indirect costs for each treatment.
Considering the four case reports, health care costs (without cost of treatment) and indirect costs per patient per year were lower with VWF with a low FVIII content than VWF/FVIII concentrates. The total health care costs (without cost of treatment) and indirect costs avoided with VWF with a low FVIII content per patient per year ranged from €2,295 to €17,530 and from €1,867 to €4,978, respectively.
VWF with a low FVIII content seems to be a cost-effective treatment option for patients with severe VWD. Although the drug cost per se is higher, the use of VWF with a low FVIII content is associated with decreased consumption of hospital resources and fewer lost working days due to bleedings and consequently with an improvement of the quality of life of the patients.
使用血管性血友病因子（VWF）/凝血因子VIII（FVIII）浓缩物进行预防是重度血管性血友病（VWD）患者的一种潜在治疗方法。据我们所知，迄今为止尚未进行过药物经济学分析以评估重度VWD治疗的经济影响。本文所呈现的分析估计了与意大利目前用于重度VWD患者长期预防的VWF/FVIII浓缩物相比，低FVIII含量VWF的成本效益比。
进行了一项成本后果分析，从意大利国家卫生服务体系和社会的角度评估重度VWD治疗的经济影响。该分析基于4例使用VWF/FVIII浓缩物和低FVIII含量VWF进行VWD长期预防的病例报告。每位患者的成本包括每种治疗的直接和间接成本。
考虑这4例病例报告，低FVIII含量VWF的每位患者每年的医疗保健成本（不包括治疗成本）和间接成本低于VWF/FVIII浓缩物。低FVIII含量VWF每位患者每年避免的总医疗保健成本（不包括治疗成本）和间接成本分别为2295欧元至17530欧元和1867欧元至4978欧元。
低FVIII含量VWF似乎是重度VWD患者具有成本效益的治疗选择。尽管药物本身成本较高，但使用低FVIII含量VWF可减少医院资源消耗，因出血导致的工作日损失减少，从而改善患者生活质量。
