目的 评价曲妥珠单抗(赫赛汀)辅助治疗与单纯化疗治疗人表皮生长因子受体-2(HER2)阳性早期乳腺癌的长期成本-效果,为广州市乳腺癌分子靶向治疗患者援助项目(PAP项目)的持续和调整提供依据。方法 构建马尔科夫(Markov)模型,建立5种马尔科夫Markov状态,模型的循环周期为1年,模拟两组患者长期生存(45年)获得的质量调整生命年(QALYs)。患者各状态的效用值和疾病转移概率来自曲妥珠单抗临床试验和已经公开发表的文献。从医保部门的角度,本研究仅考虑直接医疗成本。乳腺癌治疗成本源于广州市15家三甲医院的乳腺癌专家的问卷调查,药物及HER2检测成本由Roche制药公司提供,采用3%的贴现率对成本和健康产出进行贴现。对贴现率、药品费用等变量进行单因素敏感性分析。采用蒙特卡洛模拟进行概率敏感性分析,结果用增量成本效果散点图表示。结果 无援助项目时,曲妥珠单抗组增加1.79个QALYs,成本增加了53 301元,此时增量成本效果比(ICER)为29 731元/QALY,以2015年广州市人均GDP作为阈值时,具有成本-效果优势;有援助项目时,曲妥珠单抗组增加1.79个QALYs,直接医疗成本降低37 833元,为绝对优势方案。敏感性分析结果显示,在参数的不确定性取值变化范围内,模型具有稳健性,提示本研究结果稳定。结论 与单纯化疗相比,曲妥珠单抗辅助治疗具有更好的疗效以及经济性。广州市乳腺癌分子靶向治疗患者援助项目大幅度缓解了乳腺癌患者的疾病经济负担,值得持续和推广。
Abstract
OBJECTIVE To advocate Patient Assistant Program Projects (PAP Projects) decision-making, this study assesses the long-term cost-effectiveness of 1-year adjuvant trastuzumab therapy for women with human epidermal growth factor receptor 2 (HER2) positive early breast cancer. METHODS A Markov model tracked yearly patients’ transitions between five health states. The cycle length was 1 year and the sum was 45. From the perspective of the China health insurance system, the direct medical cost was estimated based on a survey of clinical expert panels. A discounting rate of 3% was used to discount direct medical cost and health outcomes. Utility and transition probabilities were retrieved from the HERA trial and literature. To estimate the direct medical cost, a survey of clinical expert panels was conducted. The cost of trastuzumab and HER2 test based on Roche. The key factor of the model was realized by one-way sensitivity analysis. The result of a probability sensitivity analysis conducted by Monte Carlo simulation was expressed as an incremental cost-effectiveness scatter plot. RESULTS Without PAP Projects in Guangzhou, the adjuvant trastuzumab treatment prolonged 1.79 QALYs when the cost increased ￥53 301 and the Incremental cost-effectiveness ratio (CER) was ￥29 731/ QALY, which is cost-effective based on Guangzhou's per capita GDP in 2015. With PAP Projects, the adjuvant trastuzumab treatment was totally cost-effectiveness. The sensitivity analysis demonstrated that the model was moderate. CONCLUSION One year adjuvant trastuzumab treatment is a cost-effective therapy for patients with HER-2 positive breast cancer. With PAP Projects in Guangzhou, the adjuvant trastuzumab treatment is projected to be associated with improved QALYs and reduces direct medical costs, compared with the standard chemotherapy, represents a dominant treatment option among patients with HER2-Positive Early Breast Cancer. PAP Projects in Guangzhou should be persisted and spread in China.
关键词
曲妥珠单抗 /
赫赛汀 /
早期乳腺癌 /
HER2阳性 /
成本-效果 /
PAP项目
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Key words
trastuzumab /
herceptin /
early breast cancer /
HER2-positive /
cost-effectiveness /
PAP projects
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中图分类号：
R965
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参考文献
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脚注
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基金
暨南大学“公共卫生与预防医学”一级学科师资基金(JNUPHPM0301)
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