Pharmerit International, 3068 AV Rotterdam, The Netherlands.
J Med Econ. 2013 Sep;16(9):1106-19. doi: 10.3111/13696998.2013.819357. Epub 2013 Jul 19.
Surgical resection of gastrointestinal stromal tumour (GIST) is rarely curative in patients at high risk of tumour recurrence and therefore 1 year of post-surgery adjuvant imatinib therapy has been recommended in this sub-group. Recently, adjuvant imatinib therapy administered for 3 years has been demonstrated to further increase recurrence-free survival and overall survival. The goal of this study was to assess the economic value of extending the duration of adjuvant imatinib therapy in high-risk patients in the Netherlands.
A multistate Markov model was developed to simulate how patients' clinical status after GIST excision evolves over time until death. The model structure encompassed four primary health states: free of recurrence, first GIST recurrence, second GIST recurrence, and death. Transition probabilities between the health states, data on medical care costs, and quality-of-life were obtained from published sources and from expert opinion.
The expected number of life years (or quality-adjusted life years, QALYs) was higher in the 3-year group than in the 1-year group, 8.91 (6.55) and 7.04 (5.18) years, respectively. In the 3-year and 1-year group, the expected total costs amounted to €120,195 and €79,361, of which, €74,631 (62%) and €27,619 (35%) were adjuvant therapy drug costs, respectively. The difference in health benefits, that is 1.87 life years or 1.37 QALYs, and costs, €40,835, resulted in incremental cost-effectiveness ratios (ICER) of €21,865 per life year gained, and €29,872 per QALY gained.
A limitation of the study was inherently related to the uncertainty around the predictions of RFS. Scenario analyses were conducted to test the sensitivity of different RFS predictions on the results.
Delayed recurrence due to treatment with longer-term adjuvant imatinib therapy represents a cost-effective treatment option with an ICER below the generally accepted threshold in the Netherlands.
胃肠道间质瘤(GIST)的手术切除在肿瘤复发风险高的患者中很少能达到治愈效果,因此建议这部分患者在手术后接受 1 年的辅助伊马替尼治疗。最近,辅助伊马替尼治疗 3 年的研究已经证明可以进一步提高无复发生存率和总生存率。本研究的目的是评估在荷兰高危患者中延长辅助伊马替尼治疗时间的经济价值。
采用多状态马尔可夫模型来模拟 GIST 切除后患者的临床状况随时间的演变,直至死亡。模型结构包括四个主要健康状态:无复发、首次 GIST 复发、再次 GIST 复发和死亡。健康状态之间的转移概率、医疗成本数据和生活质量数据均来自已发表的资料和专家意见。
3 年组的预期寿命(或质量调整生命年,QALY)高于 1 年组,分别为 8.91(6.55)和 7.04(5.18)年。在 3 年组和 1 年组中,预计总费用分别为 120195 欧元和 79361 欧元,其中辅助治疗药物费用分别为 74631 欧元(62%)和 27619 欧元(35%)。健康效益的差异,即 1.87 年生命或 1.37 QALY,和成本的差异,40835 欧元,导致增量成本效益比(ICER)分别为每年每获得 1 个生命 21865 欧元和每年每获得 1 个 QALY 29872 欧元。
本研究的一个局限性与无复发生存率(RFS)预测的不确定性有关。进行了情景分析以测试不同 RFS 预测对结果的敏感性。
由于接受更长时间的辅助伊马替尼治疗而延迟复发代表了一种具有成本效益的治疗选择,其增量成本效益比(ICER)低于荷兰普遍接受的阈值。