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伊布替尼作为无 17p 缺失的老年慢性淋巴细胞白血病一线治疗的成本效益。

Cost-effectiveness of ibrutinib as first-line therapy for chronic lymphocytic leukemia in older adults without deletion 17p.

机构信息

Veterans Affairs Palo Alto Health Care System, Palo Alto, CA.

Center for Primary Care and Outcomes Research/Center for Health Policy, Department of Medicine, Stanford University School of Medicine.

出版信息

Blood Adv. 2018 Aug 14;2(15):1946-1956. doi: 10.1182/bloodadvances.2017015461.

Abstract

Ibrutinib is a novel oral therapy that has shown significant efficacy as initial treatment of chronic lymphocytic leukemia (CLL). It is a high-cost continuous therapy differing from other regimens that are given for much shorter courses. Our objective was to evaluate the cost-effectiveness of ibrutinib for first-line treatment of CLL in patients older than age 65 years without a 17p deletion. We developed a semi-Markov model to analyze the cost-effectiveness of ibrutinib vs a comparator therapy from a US Medicare perspective. No direct comparison between ibrutinib and the best available treatment alternative, obinutuzumab plus chlorambucil (chemoimmunotherapy), exists. Therefore, we compared ibrutinib to a theoretical treatment alternative, which was modeled to confer the effectiveness of an inferior treatment (chlorambucil alone) and the costs and adverse events of chemoimmunotherapy, which would provide ibrutinib with the best chance of being cost-effective. Even so, the incremental cost-effectiveness ratio of ibrutinib vs the modeled comparator was $189 000 per quality-adjusted life-year (QALY) gained. To reach a willingness-to-pay threshold (WTP) of $150 000 per QALY, the monthly cost of ibrutinib would have to be at most $6800, $1700 less than the modeled cost of $8500 per month (a reduction of $20 400 per year). When the comparator efficacy is increased to more closely match that seen in trials evaluating chemoimmunotherapy, ibrutinib costs more than $262 000 per QALY gained, and the monthly cost of ibrutinib would need to be lowered to less than $5000 per month to be cost-effective. Ibrutinib is not cost-effective as initial therapy at a WTP threshold of $150 000 per QALY gained.

摘要

伊布替尼是一种新型口服治疗药物,作为慢性淋巴细胞白血病(CLL)的初始治疗,已显示出显著疗效。它是一种高成本的持续治疗方法,与其他疗程较短的方案不同。我们的目的是评估伊布替尼作为无 17p 缺失的 65 岁以上患者 CLL 一线治疗的成本效益。我们从美国医疗保险的角度开发了一个半马尔可夫模型来分析伊布替尼与对照疗法的成本效益。目前,伊布替尼与最佳治疗方案(奥滨尤妥珠单抗联合苯丁酸氮芥,化疗免疫治疗)之间没有直接比较。因此,我们将伊布替尼与一种理论上的替代治疗方案进行了比较,这种替代方案模拟了一种效果较差的治疗方案(苯丁酸氮芥单药治疗),同时模拟了化疗免疫治疗的成本和不良反应,从而为伊布替尼具有成本效益提供了最佳机会。即便如此,伊布替尼与模拟对照药物相比的增量成本效益比为每获得一个质量调整生命年(QALY)增加 189000 美元。为了达到 15 万美元/QALY 的意愿支付阈值(WTP),伊布替尼的月费用必须最多为 6800 美元,比每月 8500 美元的模型成本低 1700 美元(每年减少 20400 美元)。当对照药物的疗效提高到更接近评估化疗免疫治疗的试验中观察到的疗效时,伊布替尼的成本超过每获得一个 QALY 增加 262000 美元,而伊布替尼的月费用需要降低到每月不到 5000 美元才能具有成本效益。伊布替尼作为初始治疗方案,在 15 万美元/QALY 的 WTP 阈值下并不具有成本效益。

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