National School of Public Health, Athens, Greece.
Cancer Manag Res. 2013 Apr 10;5:37-48. doi: 10.2147/CMAR.S43373. Print 2013.
Multiple myeloma is a hematologic malignancy that incurs a substantial economic burden in care management. Since most patients with multiple myeloma eventually relapse or become refractory to current therapies (rrMM), the aim of this study was to assess the cost-effectiveness of the combination of lenalidomide-dexamethasone, relative to bortezomib alone, in patients suffering from rrMM in Greece.
An international discrete event simulation model was locally adapted to estimate differences in overall survival and treatment costs associated with the two alternative treatment options. The efficacy data utilized came from three international trials (MM-009, MM-010, APEX). Quality of life data were extracted from the published literature. Data on resource use and prices came from relevant local sources and referred to 2012. The perspective of the analysis was that of public providers. Total costs for monitoring and administration of therapy to patients, management of adverse events, and cost of medication were captured. A 3.5% discount rate was used for costs and health outcomes. A Monte Carlo simulation was used to estimate probabilistic results with 95% uncertainty intervals (UI) and a cost-effectiveness acceptability curve.
The mean number of quality-adjusted life years (QALYs) was 3.01 (95% UI 2.81-3.20) and 2.22 (95% UI 2.02-2.41) for lenalidomide-dexamethasone and bortezomib, respectively, giving an incremental gain of 0.79 (95% UI 0.49-1.06) QALYs in favor of lenalidomide-dexamethasone. The mean cost of therapy per patient was estimated at €80;77,670 (95% UI €80;76,509-€80;78,900) and €80;48,928 (95% UI €80;48,300-€80;49,556) for lenalidomide-dexamethasone and bortezomib, respectively. The incremental cost per life year gained with lenalidomide-dexamethasone was estimated at €80;29,415 (95% UI €80;23,484-€80;37,583) and the incremental cost per QALY gained at €80;38,268 (95% UI €80;27,001-€80;58,065). The probability of lenalidomide-dexamethasone being a cost-effective therapy option at a threshold three times the per capita income (€80;60,000 per QALY) was higher than 95%. The results remained constant, without altering the conclusions, under several hypothetical scenarios.
The combination of lenalidomide and dexamethasone may represent a cost-effective choice relative to bortezomib monotherapy for patients in Greece with previously treated multiple myeloma.
多发性骨髓瘤是一种血液系统恶性肿瘤，在治疗管理方面会带来巨大的经济负担。由于大多数多发性骨髓瘤患者最终会复发或对当前的治疗方法产生耐药（rrMM），因此本研究旨在评估来那度胺-地塞米松联合治疗与硼替佐米单药治疗在希腊 rrMM 患者中的成本效益。
对国际离散事件模拟模型进行了本地化调整，以评估两种替代治疗方案的总生存和治疗成本差异。使用的疗效数据来自三项国际试验（MM-009、MM-010、APEX）。从已发表的文献中提取生活质量数据。资源利用和价格数据来自相关的本地来源，并参考了 2012 年的数据。分析的角度是公共提供者。监测和管理患者治疗、不良事件管理以及药物治疗成本都被计入在内。采用 3.5%的贴现率来计算成本和健康结果。采用蒙特卡罗模拟来估计具有 95%不确定性区间（UI）和成本效益可接受性曲线的概率结果。
来那度胺-地塞米松组的平均质量调整生命年（QALY）为 3.01（95%UI 2.81-3.20），硼替佐米组为 2.22（95%UI 2.02-2.41），来那度胺-地塞米松组比硼替佐米组有 0.79（95%UI 0.49-1.06）的 QALY 增量获益。每位患者的治疗平均成本估计为 8077670 欧元（95%UI 8076509-8078900 欧元）和 8048928 欧元（95%UI 8048300-8048956 欧元），分别用于来那度胺-地塞米松和硼替佐米。使用来那度胺-地塞米松的增量成本每获得一年生命的费用估计为 8029415 欧元（95%UI 8023484-8037583 欧元），每获得一个 QALY 的增量成本为 8038268 欧元（95%UI 8027001-8058065 欧元）。来那度胺-地塞米松作为一种成本效益治疗选择的概率高于 95%，在阈值为人均收入的三倍（每 QALY 806000 欧元）时，该疗法的成本效益更高。在几种假设情况下，结果仍然保持不变，没有改变结论。
对于希腊既往治疗过的多发性骨髓瘤患者，来那度胺联合地塞米松治疗可能是一种比硼替佐米单药治疗更具成本效益的选择。
