阿扎胞苷与地西他滨治疗骨髓增生异常综合征的成本-效用分析

Cost-utility analysis of azacitidine versus decitabine in the treatment of myelodysplastic syndrome

目的从我国卫生体系角度出发评价阿扎胞苷与地西他滨治疗骨髓增生异常综合征(MDS)的经济性,为临床合理用药提供参考。方法基于一项多中心回顾性队列研究(NCT01409070)的数据,构建Markov模型,模型模拟时限为10年,循环周期为28 d,以质量调整生命年(QALYs)作为健康产出指标并计算增量成本-效用比(ICUR),评价阿扎胞苷与地西他滨治疗MDS的经济性,采用单因素敏感性分析和概率敏感性分析验证基础分析结果的稳定性。结果基础分析结果显示,与地西他滨治疗方案相比,阿扎胞苷治疗方案的总成本降低281185.46元,且效用增加0.17 QALYs,阿扎胞苷治疗方案为绝对优势方案。单因素敏感性分析结果显示,无进展生存状态效用值、贴现率和地西他滨药费对结果的影响相对较大。概率敏感性分析结果提示,在3倍我国2021年人均国内生产总值为意愿支付阈值范围内,阿扎胞苷治疗方案始终具有经济性。结论从我国卫生体系角度出发,与地西他滨相比,阿扎胞苷治疗MDS更具成本-效用优势。

Objective To evaluate the economics of azacitidine versus decitabine in the treatment of myelodysplastic syndrome(MDS)from the perspective of health system in China,and provide references for clinical drug selection.Methods A Markov model was constructed based on the data of a multi-center retrospective cohort study(NCT01409070),with a model simulation time limit of 10 years and the cycle period of 28 days.The quality-adjusted life years(QALYs)was used as health output index and incremental cost-utility ratio(ICUR)was calculated to evaluate the economics of azacitidine versus decitabine in the treatment of MDS.One-way sensitivity analysis and probabilistic sensitivity analysis were adopted to examine the robustness of the model simulation results.Results The results of basic analysis showed that compared with decitabine group,the total cost of azacitidine group reduced by 281185.46 yuan and the utility increased by 0.17 QALYs.Azacitidine therapeutic regimen was the absolute dominance plan.One-way sensitivity analysis showed that progression-free survival state utility value,discount rate and decitabine cost had greater influence on the results.The results of probabilistic sensitivity analysis suggested that azacinidine therapy was always economical within the willingness to pay threshold range of 3 times Chinese per capita gross domestic product in 2021.Conclusion From the perspective of health system in China,azacitidine has more cost-utility advantages than decitabine in the treatment of MDS.