卡非佐米不同剂量方案治疗复发/难治多发性骨髓瘤的成本-效用分析

    Cost-utility Analysis of Different Dosage Regimens of Carfilzomib in the Treatment of Relapsed/ Refractory Multiple Myeloma

    • 摘要:
      目的  从中国卫生体系角度评价卡非佐米不同剂量方案治疗复发/难治多发性骨髓瘤(relapsed/refractory multiple myeloma,RRMM)的经济性。
      方法 利用临床试验数据和相关文献数据构建包含肿瘤“无进展”“进展”“死亡”三状态的Markov模型。循环周期为1个月,模拟RRMM患者10年内的疾病进展状况,成本和效用数据均采用5%的贴现率进行贴现处理。以增量成本-效用比(incremental cost-utility ratio,ICUR)为评价指标,意愿支付阈值(willingness-to-pay,WTP)设为 2024 年中国1~3 倍人均国内生产总值(gross domestic product,GDP)(95 749 ~287 247元),并采用单因素敏感性分析评价模型参数变化时模型结果的稳定性。
      结果 基础分析结果显示,Kd27方案(卡非佐米27 mg·m−2,每周2次+地塞米松,238例)和Kd70方案(卡非佐米70 mg·m−2,每周1次+地塞米松,240例)属于高成本、高健康获益方案,与Kd56方案(卡非佐米56 mg·m−2,每周2次+地塞米松,464例)相比的ICUR值分别为Kd27:211 201.66元·质量调整生命年(quality-adjusted life years,QALYs)−1,Kd70:252 181.96元·QALYs−1。单因素敏感性分析结果表明疾病无进展生存期效用值是影响整体治疗经济性结果的关键因素,仅当该参数取上限值时,ICUR高于WTP,其余所有参数在上下限全区间波动时, ICUR 均低于 WTP,表明本研究模型分析结论具备较高稳健性。
      结论 在中国医保体系下,卡非佐米不同剂量方案治疗RRMM,与Kd56方案相比,Kd27方案、Kd70方案有效性和经济性具有一定优势,且Kd27方案成本-效用最佳。患者可根据自身需要和医师建议选择合适治疗方案。

       

      Abstract:
      OBJECTIVE  To evaluate the economics of different dosage regimens of carfilzomib in the treatment of relapsed/refractory multiple myeloma(RRMM) from the perspective of the Chinese healthcare system.
      METHODS  A Markov model encompassing three health states including “progression-free” “progressed” and “dead”, which was constructed using clinical trial data and relevant literature. The cycle length was set at one month, simulating the disease progression of RRMM patients over a 10-year period. Cost and utility data were discounted at a rate of 5%. The incremental cost-utility ratio(ICUR) was used as the evaluation metric, with the willingness-to-pay(WTP) threshold set at 1−3 times the per capita gross domestic product(GDP) of China in 2024(ranging from 95 749 yuan to 287 247 yuan). One-way sensitivity analysis was conducted to assess the stability of the model results when parameters were varied.
      RESULTS  The base-case analysis revealed that The Kd27 regimen(carfilzomib 27 mg·m−2 twice weekly+dexamethasone, n=238) and the Kd70 regimen(carfilzomib 70 mg·m−2 once weekly+dexamethasone, n=240) were high cost, high health benefit strategies, compared to the Kd56 regimen(carfilzomib 56 mg·m−2 twice weekly+dexamethasone, n=464). The ICUR values for Kd27 and Kd70 were 211 201.66 yuan·(quality-adjusted life years, QALYs)−1 and 252 181.96 yuan·QALYs−1, respectively. One-way sensitivity analysis indicated that the utility value during progressive free survival was a key factor influencing the overall economic evaluation results. The ICUR exceeded the WTP threshold only at the upper bound of this parameter, while remaining below it across all other parameter ranges, confirming the robustness of our model-based conclusions.
      CONCLUSION  Within the Chinese healthcare insurance system, different doses of caffezomib in the treatment of RRMM, compared with Kd56, Kd27 and Kd70 have certain advantages in effectiveness and economy, and KD27 has the best cost-effectiveness. Patients may choose an appropriate treatment regimen based on their individual needs and doctor’s recommendations.

       

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