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接受靶向治疗的转移性肾细胞癌患者的真实世界成本和结局:一项来自法国健康保险数据库的队列研究

Real-world costs and outcomes in metastatic renal cell carcinoma patients treated with targeted therapies: a cohort study from the French health insurance database.

作者信息

Maroun Rana, Fleury Laetitia, Nachbaur Gaelle, Maunoury Franck, Vanhille Jean-Louis, Durand-Zaleski Isabelle

机构信息

a GlaxoSmithKline, Health Outcomes Research , Marly le Roi , France.

b INSERM, ECEVE, UMR 1123 , Paris , France.

出版信息

Curr Med Res Opin. 2017 Oct;33(10):1755-1762. doi: 10.1080/03007995.2017.1360850. Epub 2017 Aug 7.

Abstract

OBJECTIVES

The objective of this study was to describe treatment patterns, survival, healthcare use and costs in patients with metastatic renal cell carcinoma (mRCC) in a real-world setting.

RESEARCH DESIGN AND METHODS

We used the National Health Insurance (NHI) claims database for the Ile-de-France region to perform a retrospective cohort analysis of patients with mRCC treated by a first-line targeted therapy. Treatment naïve patients were identified combining the 10th revision of the International Classification of Diseases (ICD-10) codes (C64 & C77-C79) and a first prescription of targeted therapies. Descriptive analyses were performed on treatment patterns and patients' characteristics. Progression free survival (PFS) and overall survival (OS) were determined using Kaplan-Meier actuarial survival analysis. All healthcare resource use and costs were estimated on a per patient per month (PPPM) basis (€2016).

RESULTS

A total of 327 treatment naïve patients with mRCC were included. Median follow-up was 13.4 months. Sunitinib accounted for 73% of first-line treatments. The most frequently observed treatment sequence for the first two lines was sunitinib-everolimus (16%; n = 137) and for the first three lines sunitinib-everolimus-axitinib (20%; n = 49). First-line PFS for sunitinib, everolimus, pazopanib, sorafenib and other was 8.7, 6.2, 10.7, 5.7 and 11.2 months, respectively. Median OS for patients treated by first-line sunitinib, everolimus, pazopanib, sorafenib and other was respectively 14.7, 8.1, 21.1, 8.9 and 14.0 months. From the NHI's perspective, the mean PPPM was €5546. The average PPPM in pre-progression was €5597 compared to €5541 beyond progression of the disease. Oral targeted therapies accounted for 53% of the total PPPM.

CONCLUSION

This descriptive study showed that the economic burden of mRCC is substantial with oral targeted therapies accounting for 53% of the PPPM. OS and PFS in real life are poorer than observed in clinical trials.

摘要

目的

本研究的目的是描述真实世界中转移性肾细胞癌(mRCC)患者的治疗模式、生存率、医疗资源使用情况及成本。

研究设计与方法

我们使用法国巴黎大区的国民健康保险(NHI)理赔数据库,对接受一线靶向治疗的mRCC患者进行回顾性队列分析。通过结合国际疾病分类第10版(ICD-10)编码(C64及C77 - C79)和首次靶向治疗处方,识别出未接受过治疗的患者。对治疗模式和患者特征进行描述性分析。使用Kaplan-Meier精算生存分析确定无进展生存期(PFS)和总生存期(OS)。所有医疗资源使用情况和成本均按每位患者每月(PPPM)进行估算(2016年欧元)。

结果

共纳入327例未接受过治疗的mRCC患者。中位随访时间为13.4个月。舒尼替尼占一线治疗的73%。前两线最常观察到的治疗顺序是舒尼替尼 - 依维莫司(16%;n = 137),前三线是舒尼替尼 - 依维莫司 - 阿昔替尼(20%;n = 49)。舒尼替尼、依维莫司、帕唑帕尼、索拉非尼及其他药物的一线PFS分别为8.7、6.2、10.7、5.7和11.2个月。接受一线舒尼替尼、依维莫司、帕唑帕尼、索拉非尼及其他药物治疗的患者的中位OS分别为14.7、8.1、21.1、8.9和14.0个月。从NHI的角度来看,平均PPPM为5546欧元。疾病进展前的平均PPPM为5597欧元,疾病进展后的平均PPPM为5541欧元。口服靶向治疗占总PPPM的53%。

结论

这项描述性研究表明,mRCC的经济负担巨大,口服靶向治疗占PPPM的53%。现实生活中的OS和PFS比临床试验中观察到的情况更差。

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