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一种类似BRCA1检测以发现对高剂量烷化剂化疗有反应的三阴性乳腺癌的早期成本效益分析。

Early stage cost-effectiveness analysis of a BRCA1-like test to detect triple negative breast cancers responsive to high dose alkylating chemotherapy.

作者信息

Miquel-Cases Anna, Steuten Lotte M G, Retèl Valesca P, van Harten Wim H

机构信息

Netherlands Cancer Institute-Antoni van Leeuwenhoek Hospital (NKI-AVL), Department of Psychosocial Research and Epidemiology, Plesmanlaan 121, 1066 CX, Amsterdam, The Netherlands.

Hutchinson Institute for Cancer Outcomes Research, Fred Hutchinson Cancer Research Center, Seattle, USA.

出版信息

Breast. 2015 Aug;24(4):397-405. doi: 10.1016/j.breast.2015.03.002. Epub 2015 Apr 28.

Abstract

PURPOSE

Triple negative breast cancers (TNBC) with a BRCA1-like profile may benefit from high dose alkylating chemotherapy (HDAC). This study examines whether BRCA1-like testing to target effective HDAC in TNBC patients can be more cost-effective than treating all patients with standard chemotherapy. Additionally, we estimated the minimum required prevalence of BRCA1-like and the required positive predictive value (PPV) for a BRCA1-like test to become cost-effective.

METHODS

Our Markov model compared 1) the incremental costs; 2) the incremental number of respondents; 3) the incremental number of Quality Adjusted Life Years (QALYs); and 4) the incremental cost-effectiveness ratio (ICER) of treating TNBC women with personalized HDAC based on BRCA1-like testing vs. standard chemotherapy, from a Dutch societal perspective and a 20-year time horizon, using probabilistic sensitivity analysis. Furthermore, we performed one-way sensitivity analysis (SA) to all model parameters, and two-way SA to prevalence and PPV. Data were obtained from a current trial (NCT01057069), published literature and expert opinions.

RESULTS

BRCA1-like testing to target effective HDAC would presently not be cost-effective at a willingness-to-pay threshold of €80.000/QALY (€81.981/QALY). SAs show that PPV drives the ICER changes. Lower bounds for the prevalence and the PPV were found to be 58.5% and 73.0% respectively.

CONCLUSION

BRCA1-like testing to target effective HDAC treatment in TNBC patients is currently not cost-effective at a willingness-to-pay of €80.000/QALY, but it can be when a minimum PPV of 73% is obtained in clinical practice. This information can help test developers and clinicians in decisions on further research and development of BRCA1-like tests.

摘要

目的

具有BRCA1样特征的三阴性乳腺癌(TNBC)患者可能从高剂量烷化剂化疗(HDAC)中获益。本研究旨在探讨针对TNBC患者进行BRCA1样检测以确定有效的HDAC治疗是否比用标准化疗治疗所有患者更具成本效益。此外,我们估计了BRCA1样特征的最低所需患病率以及BRCA1样检测具有成本效益所需的阳性预测值(PPV)。

方法

我们的马尔可夫模型从荷兰社会视角和20年时间范围,使用概率敏感性分析,比较了以下方面:1)增量成本;2)应答者增量数量;3)质量调整生命年(QALY)增量数量;4)基于BRCA1样检测的个性化HDAC治疗TNBC女性与标准化疗的增量成本效益比(ICER)。此外,我们对所有模型参数进行了单向敏感性分析(SA),并对患病率和PPV进行了双向SA。数据来自当前试验(NCT01057069)、已发表的文献和专家意见。

结果

在支付意愿阈值为80,000欧元/QALY(81,981欧元/QALY)时,针对有效HDAC的BRCA1样检测目前不具有成本效益。敏感性分析表明PPV驱动ICER变化。发现患病率和PPV的下限分别为58.5%和73.0%。

结论

在支付意愿为80,000欧元/QALY时,针对TNBC患者进行BRCA1样检测以确定有效的HDAC治疗目前不具有成本效益,但在临床实践中获得至少73%的PPV时则可能具有成本效益。该信息可帮助检测开发者和临床医生在BRCA1样检测的进一步研发决策中提供参考。

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