Earnshaw Stephanie R, Joshi Ashish V, Wilson Michele R, Rosand Jonathan
RTI Health Solutions, Research Triangle Park, NC, USA.
Stroke. 2006 Nov;37(11):2751-8. doi: 10.1161/01.STR.0000246611.21999.5d. Epub 2006 Sep 28.
Intracerebral hemorrhage (ICH) is among the most costly and debilitating forms of stroke. Results from a recent Phase IIb clinical trial demonstrate that administration of recombinant activated factor VII (rFVIIa) reduces ICH mortality and improves functional outcome. In the current analysis, we examine the cost-effectiveness of early treatment with rFVIIa for ICH in the United States.
A decision-analytic model was developed to estimate the lifetime costs and outcomes associated with rFVIIa treatment at doses of 40, 80 and 160 microg/kg compared with current standard of care in treating ICH, from a US third-party payer perspective. The patient population was similar to that of the Phase IIb clinical trial. Model structure and inputs were obtained from published literature, clinical trial data, claims databases, and expert opinion. All costs are presented in 2005 US dollars. Outcomes included incremental cost per life-year (LY) saved and incremental cost per quality-adjusted life-year (QALY) gained. Costs and outcomes were discounted at 3% annually. Univariate and multivariate sensitivity analyses were conducted to assess model robustness.
Compared with standard care, treatment with rFVIIa 40 microg/kg, and 160 microg/kg results in total lifetime cost-effectiveness ratios of 6308 dollars/QALY and 3152 dollars/QALY, respectively. Treatment with rFVIIa 80 microg/kg was found to be cost saving and a gain of 1.67 QALYs is achieved over a patient's lifetime. These results are robust to changes in input parameters.
Treatment of ICH with rFVIIa 40 microg/kg and 160 microg/kg appears to be cost-effective (<or=50,000 dollars/QALY). At the 80 microg/kg dose, rFVIIa was not only cost-effective, but also cost saving.
脑出血（ICH）是最昂贵且使人衰弱的中风形式之一。最近一项IIb期临床试验结果表明，给予重组活化因子VII（rFVIIa）可降低脑出血死亡率并改善功能转归。在当前分析中，我们研究了在美国早期使用rFVIIa治疗脑出血的成本效益。
建立了一个决策分析模型，从美国第三方支付者的角度，估计与剂量为40、80和160μg/kg的rFVIIa治疗相关的终身成本和转归，与当前治疗脑出血的标准治疗方法进行比较。患者群体与IIb期临床试验的相似。模型结构和输入数据来自已发表的文献、临床试验数据、索赔数据库和专家意见。所有成本均以2005年美元表示。转归包括每挽救一个生命年（LY）的增量成本和每获得一个质量调整生命年（QALY）的增量成本。成本和转归按每年3%进行贴现。进行单因素和多因素敏感性分析以评估模型的稳健性。
与标准治疗相比，40μg/kg和160μg/kg的rFVIIa治疗的终身总成本效益比分别为6308美元/QALY和3152美元/QALY。发现80μg/kg的rFVIIa治疗具有成本节约效益，患者一生可获得1.67个QALY。这些结果对输入参数的变化具有稳健性。
40μg/kg和160μg/kg的rFVIIa治疗脑出血似乎具有成本效益（≤50,000美元/QALY）。在80μg/kg剂量下，rFVIIa不仅具有成本效益，而且还具有成本节约效益。
