泊沙康唑与伊曲康唑预防骨髓增生异常综合征/急性髓系白血病患者侵袭性真菌病的成本-效果分析

Cost-effectiveness of posaconazole versus itraconazole for invasive fungal disease preventionamong myelodysplasia syndrome or acute myeloid leukemia patients

目的:开展泊沙康唑对比伊曲康唑用于预防骨髓增生异常综合征/急性髓系白血病患者发生侵袭性真菌病的成本-效果分析,为临床合理用药、药品费用不合理增长及医保药品目录的遴选提供决策依据。方法:基于医疗卫生体系角度,构建决策树模型及Markov模型,比较泊沙康唑和伊曲康唑用于预防侵袭性真菌病的短期和长期的经济性;短期健康产出为侵袭性真菌病的发生率,长期健康产出为患者所获得的生命年和质量调整生命年,成本数据来源于2019年中国真实医疗环境价格及文献研究。进行敏感性分析以验证基础分析结果的稳健性。结果:与伊曲康唑相比,泊沙康唑在短期模拟的100 d成本更高(10992元vs.8038元),但预防效果更好(侵袭性真菌病发生率4.6%vs.11.1%)。在长期的患者全生命周期模拟中获得的生命年和质量调整生命年更多(2.949 LYs vs.2.719 LYs,2.096 QALYs vs.1.932 QALYs),其增量成本-效果比为18030元/QALY,远低于2019年中国社会意愿支付阈值(212676元/QALY)。敏感性分析结果与基础分析结果一致。结论:在中国,泊沙康唑相比伊曲康唑用于预防骨髓增生异常综合征/急性髓系白血病患者发生侵袭性真菌病的经济性更好。

OBJECTIVE To examine the cost-effectiveness of posaconazole versus itraconazole for invasive fungal disease(IFD)prevention among myelodysplasia syndrome or acute myeloid leukemia patients,and to provide evidence for decision-making in the selection of antifungal drugs,the selection of medical insurance catalogue,and the control of increasing medication costs.METHODS Based on the healthcare system perspective,a decision tree model and a Markov model were conducted to compare the short-term cost-effectiveness and long-term cost-utility of posaconazoleand itraconazole for IFD prevention.The short-term health outcome was the incidence of invasive mycosis;the long-term health outcome was the life years obtained by patients and the quality-adjusted life years,and the cost data were obtained from the 2019 real medical environment price and literature study in China.Sensitivity analyses were performed to verify the robustness of the results of the base analysis.RESULTS In the short-term model(100 days),posaconazole was more expensive(10992 yuan vs.8038 yuan)and more effective(IFD 4.6%vs.11.1%)compared to itraconazole.More life years and quality-adjusted life years were obtained in the long-term patient full life cycle simulation(2.949 LYs vs.2.719 LYs,2.096 QALYs vs.1.932 QALYs),and its incremental cost-effectiveness ratio was 18030 yuan/QALY,which is much lower than the 2019 threshold for social willingness to pay in China(212676 yuan/QALY).The results of sensitivity analysis were consistent with the results of basic analysis.CONCLUSION Posaconazole is more economical than itraconazole for the prevention of invasive mycosis in patients with myelodysplastic syndrome/acute myelogenous leukemia in China.