慢性髓系白血病患者一线TKI策略的成本-效果：追求无治疗缓解和剂量减少。

IF 4.9 2区医学 Q1 ECONOMICS

Value in Health Pub Date : 2025-02-01 DOI:10.1016/j.jval.2024.12.005

Sanne J.J.P.M. Metsemakers MSc , Rosella P.M.G. Hermens PhD , Geneviève I.C.G. Ector PhD , Nicole M.A. Blijlevens PhD , Tim M. Govers PhD

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引用次数: 0

摘要

目的：慢性髓性白血病（CML）的治疗现在包括剂量减少（DR）和无治疗缓解（TFR）。评估终身处方昂贵的酪氨酸激酶抑制剂（TKIs）治疗CML的成本效益至关重要。先前的成本效益评估表明，伊马替尼是有利的一线TKI。其中一些评估涉及TFR，但不涉及DR，也不涉及老化和即将到期的第二代（2G） tki。本研究评估了包括这些因素在内的一线tki治疗CML患者的成本效益。方法：采用马尔可夫模型评估17种健康状态下一线tki治疗新诊断CML患者的成本-效果。转换概率、成本和效用来源于文献数据。计算增量成本-效果比。进行敏感性分析和模型验证。结果：在包括TFR和dr的模型中，尼洛替尼最有效（20.13 QALYs），伊马替尼最无效（17.25 QALYs），伊马替尼优于达沙替尼（89.80%）、尼洛替尼（62.70%）和博舒替尼（78.40%），WTP为80,000欧元/QALY。没有TFR和DR，产生的qaly较少。对于70岁的患者，与达沙替尼、尼洛替尼和博舒替尼相比，伊马替尼具有较高的成本效益。尼罗替尼的2GTKI成本降低了50%，与伊马替尼（98.40%）、达沙替尼（94.80%）和博舒替尼（68.90%）相比，尼罗替尼被认为具有成本效益。结论：研究结果表明，2GTKIs在产生QALYs方面更有效，包括老年（70岁）患者。考虑到目前TKI的价格，伊马替尼仍然具有成本效益。在cml管理中加入DR和TFR可以产生更多的qaly。从预计的2个gtki专利到期中减少的成本将大大提高其成本效益。研究结果可能会在专利到期后为2gtki的成本讨论提供信息，从而有可能扩大其在全球的可用性。研究结果还强调了在cml管理中以TFR和DR为目标的重要性。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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The Cost-Effectiveness of Frontline Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia: In Pursuit of Treatment-Free Remission and Dose Reduction

Objectives

The management of chronic myeloid leukemia (CML) now includes dose reduction (DR) and treatment-free remission (TFR). Evaluating the cost-effectiveness of lifelong-prescribed expensive tyrosine kinase inhibitors (TKIs) for CML is crucial. Prior cost-effectiveness evaluations state that imatinib is the favorable frontline TKI. Some of these evaluations address TFR, but not DR, nor aging and second-generation (2G)-TKIs upcoming patent expirations. This study evaluates the cost-effectiveness of frontline TKIs for CML patients including these factors.

Methods

This Markov model evaluates the cost-effectiveness of frontline TKIs for newly diagnosed patients with CML using 17 health states. Transition probabilities, costs, and utilities were derived from literature data. Incremental cost-effectiveness ratios were calculated. Sensitivity analysis and model validation were conducted.

Results

Nilotinib is most effective (20.13 quality-adjusted life-years [QALYs]) and imatinib is least effective (17.25 QALYs) for the model including TFR and DR. Imatinib was favored over dasatinib (89.80%), nilotinib (62.70%), and bosutinib (78.40%), at a willingness-to-pay threshold of €80 000 per QALY. Without TFR and DR, fewer QALYs were generated. For patients at the age of 70 years, imatinib has a high probability of being more cost-effective than dasatinib, nilotinib, and bosutinib. With 50% 2GTKI cost reductions, nilotinib is considered more cost-effective compared with imatinib (98.40%), dasatinib (94.80%), and bosutinib (68.90%).

Conclusions

The findings indicate that 2GTKIs are more effective in generating QALYs, including for older (age >70 years) patients. Given the current TKI prices, imatinib remains cost-effective. Including DR and TFR in CML management generates more QALYs. Cost reductions from expected 2GTKIs patent expirations will greatly increase their cost-effectiveness. Results may inform 2GTKIs cost discussions after patent expiration, potentially broadening global availability. The findings also emphasize the importance of aiming for TFR and DR in CML management.

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来源期刊

Value in Health 医学-卫生保健

CiteScore

6.90

自引率

6.70%

发文量

3064

审稿时长

3-8 weeks

期刊介绍： Value in Health contains original research articles for pharmacoeconomics, health economics, and outcomes research (clinical, economic, and patient-reported outcomes/preference-based research), as well as conceptual and health policy articles that provide valuable information for health care decision-makers as well as the research community. As the official journal of ISPOR, Value in Health provides a forum for researchers, as well as health care decision-makers to translate outcomes research into health care decisions.