【摘要】 目的:评估尼洛替尼一线治疗慢性髓性白血病(CML),相比于伊马替尼而言,对疾病管理费用的影响,以期为医疗保障及临床应用决策提供实证参考。方法:本研究应用Markov模型构建决策分析框架,研究跨度为10年。患者短期临床效果数据来自于国内真实世界数据,其余疗效数据主要来源于国际多中心临床试验,费用数据来源于江苏、河南、四川、河北共4家CML联盟重点医院的平均收费标准。结果 :尼洛替尼组每位患者的年平均治疗费用为69,889元,显著低于伊马替尼组的82,057元,降低约12,168元(14.83%)费用。费用节约有两个主要来源,一是有一定比例患者实现无治疗缓解(TFR),二是患者延缓进入二线治疗。敏感性分析显示该结果具有稳定性。结论 :尼洛替尼作为新诊断CML患者一线用药方案,临床疗效优于伊马替尼,能够延缓患者进入二线,部分患者实现功能性治愈,可以明显节约我国CML疾病管理费用。更多还原

【Abstract】 Objective: To evaluate the impact on disease management cost of introducing Nilotinib as first line treatment for Chronic Myelogenous Leukemia(CML) in China, compared with that of Imatinib, with a view to providing reference for medical insurance and clinical decision-making. Methods: A Markov-based decision-analytic framework was constructed to estimate the treatment cost in the following 10 years. Short-term eff ectiveness data was extracted from Chinese real world data, and other outcomes data were mainly extracted from international multicenter RCTs. Costs data was estimated basing on the average standard price of 4 CML-union centered hospitals in China. Results: The average treatment cost of Nilotinib group was RMB 69,889 per patient per year, which is significantly lower than that of Imatinib group(RMB 82,057 per patient per year), saving RMB 12,168(14.83%). There are two reasons for cost savings: one is that some patients in Nilotinib group achieved treatmentfree remission(TFR), and the other is that patients in Nilotinib group delayed the second line treatment timing. Sensitivity analysis suggested that the result was robust. Conclusions: Treating newly diagnosed CML patients with Nilotinib as first-line regimen is cost-saving in disease management cost in China since Nilotinib is superior to Imatinib in clinical efficacy.更多还原