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目的:比较使用重组活化凝血因子Ⅶ(r F Ⅶa)和致活的前凝血酶元复合浓缩物(APCC)对具有抗体的血友病患者轻-中度出血3种家庭治疗方案的成本效果。方法:用基于文献的决策分析模型,用来比较rF Ⅶa-rF Ⅶa-rF Ⅶ a、APCC-rFⅦa-rFⅦa和APCC-APCC-rFⅦa3种治疗方案。每个治疗方案都分为3个级别,一级治疗无效则使用二级治疗,二级治疗也也无效则使用三级治疗。使用不久前美国已发表的分析文献中的专家观点来建立模型并录入基础数据。从已发表文献中获得r F Ⅶa和APCC控制出血的百分数。基础数据模型中的药品成本(2005年美国标准)根据平均批发价确定。使用单变量分析和概率敏感度分析(二级Monte Carlo模拟法)分析疗效、再次出血率、患者体重和给药剂量来探知模型耐用性。结果:在基础数据分析中,rFⅦa-rFⅦa-rFⅦa方案解决出血问题的成本是28 076美元。使用APCC-r F Ⅶ a-rF Ⅶa方案的平均成本是30 883美元,而APCC-APCC-rFⅧa方案的成本是更高,平均需要32 150美元。仅使用r FⅦa的治疗方案通过避免二级和三级治疗而抵销了整体成本。通过概率灵敏度分析,超过68％的概率表明,全程治疗中仅使用r FⅦa方法的花费最少。结论:对轻-中度出血治疗的经济学思考应向初级治疗外扩展,进一步考虑到重复出血和多级治疗方案失败的因素。在大多数病例中,仅使用r F Ⅶ a的治疗方案是最经济的选择。
PURPOSE: To compare the cost-effectiveness of three home-based regimens for mild to moderate bleeding in patients with hemophilia with antibodies using recombinant activated coagulation factor Ⅶ (rVVIIa) and an activated pro-thrombin complex concentrate (APCC). METHODS: A literature-based decision analysis model was used to compare three regimens of rF Ⅶa-rF Ⅶa-rF Ⅶ a, APCC-rFⅦa-rFⅦa and APCC-APCC-rFⅦa. Each treatment plan is divided into three levels, the first level of treatment is invalid, the second level of treatment is used, and the second level of treatment is also ineffective, with the third level of treatment. Use the recent expert opinion from the published analytical literature in the United States to establish a model and enter basic data. The percentage of rF Ⅶa and APCC-controlled bleeding was obtained from published literature. The cost of medicines in the underlying data model (2005 US standards) is based on the average wholesale price. Univariate analysis and probability sensitivity analysis (two Monte Carlo simulation) analysis of efficacy, rebleeding rate, patient weight and dose to detect model durability. RESULTS: The cost of the rFVIIa-rFVIIa-rFVIIa regimen in resolving bleeding problems was $ 28,076 in the underlying data analysis. The average cost of using the APCC-rFVII-rFVIIa regimen was $ 30,883, whereas the cost of the APCC-APCC-rFVIIIa regimen was higher, with an average cost of $ 32,150. Treatment regimens using only rFVIIa offset overall costs by avoiding secondary and tertiary treatment. Probabilistic sensitivity analysis shows that a probability of over 68% indicates that the only use of the rVIIa method is minimal at full course of treatment. CONCLUSIONS: The economic thinking on the treatment of mild-to-moderate bleeding should be extended beyond primary treatment to further account for the failure of repeated bleeding and multi-stage treatment regimens. In most cases, treatment with rFVIIa alone is the most economical option.