免疫抑制疗法与造血干细胞移植治疗成人获得性重型再生障碍性贫血的成本-效果分析

Immunosuppressive therapy versus hematopoietic stem cell transplantation in patients with acquired severe aplastic anemia:a cost-effectiveness analysis

目的分析免疫抑制疗法(IST)与造血干细胞移植(HSCT)治疗成人获得性重型再生障碍性贫血(SAA)的成本-效果。方法通过文献检索获得不同病情状态之间转换的概率,从复旦大学附属华山医院及苏州大学附属第一医院获得SAA患者首次治疗及后续随访花费的中位值。采用卫生经济学的方法建立马尔科夫模型,循环状态模拟运行30年获得相应的增量成本-效果比,比较HSCT和IST治疗不同年龄分组(18~35岁组和35~50岁组)SAA患者的成本-效果。结果18~35岁组患者应用HSCT相对于IST的增量成本效果比(ICER)为14054.19美元/质量调整生命年(QALY),小于意愿支付值(WTP)25397.57美元;而对于35~50岁组患者应用HSCT组相对于IST组的QALY增量为-3.24 QALYs,成本增量为72009.35美元。结论18~35岁获得性SAA患者更推荐选择HSCT,而35~50岁获得性SAA患者更建议选择IST。

Objective To analyze the cost-effectiveness of hematopoietic stem cell transplantation(HSCT)and immunosuppressive therapy(IST)in patients of severe aplastic anemia(SAA).Methods The probability of transition between different disease states was obtained through literature search.The median values of SAA patients’first treatment and follow-up were obtained from Huashan Hospital of Fudan University and First Hospital of Suzhou University.A cost-effectiveness analysis was conducted by a Markov model compared IST with HSCT in age-stratified patients with SAA(18 to 35 years group and 35 to 50 years group).The model rab for virtual 30 years and worked out the incremental cost-effectiveness ratio.Results In patients aged 18 to 35 years,the ICER of HSCT to IST was$14054.19/quality-adjusted life years(QALY),which was less than the willingness to pay(WTP)value of$25,397.57/QALY.In patients aged 35 to 50 years,the QALY change of the HSCT group relative to the IST group was-3.24 QALYs,and the cost increase was$72009.35.Conclusion HSCT is more recommended for SAA patients aged 18 to 35,and IST is more recommended for SAA patients aged 35 to 50.