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患者将毒性与二线治疗晚期肾细胞癌的无进展生存期进行权衡。

Patients rank toxicity against progression free survival in second-line treatment of advanced renal cell carcinoma.

机构信息

Norris Comprehensive Cancer Center, University of Southern California, Los Angeles, CA 90033, USA.

出版信息

J Med Econ. 2012;15(6):1139-48. doi: 10.3111/13696998.2012.708689. Epub 2012 Jul 18.

Abstract

BACKGROUND

The aims of this study were to quantify and contrast patient preferences between second-line advanced renal cell carcinoma (RCC) medication profiles and their associated benefits and toxicities, and to help frame the doctor-patient discussion about selecting appropriate RCC therapies.

RESEARCH DESIGN AND METHODS

Adult residents of the US with a diagnosis of RCC completed a Web-enabled choice-format conjoint survey consisting of a series of 10 treatment-choice questions, each of which included a pair of hypothetical RCC medication profiles. Each profile was described by various medication attributes (features or outcomes) with varying levels. The attributes included efficacy (progression-free survival [PFS]), tolerability (fatigue, stomach problems, mucositis or stomatitis, hand-foot syndrome [HFS]), serious but rare adverse events (pneumonitis, hepatic impairment), and mode of administration. Treatment-choice questions were based on an experimental design with known statistical properties. Random-parameters logit regression was used to estimate relative preference weights for each attribute level. Benefit equivalent measures (additional months of PFS in exchange for toxicities) were also calculated.

RESULTS

Of the 272 patients who completed the survey, the majority were female (53%), white (92%), and had at least a college degree (66%). The mean age was 57 years (standard deviation: 10 years). Over the range of attributes and attribute levels included in the survey, PFS was the most important attribute, followed by fatigue, stomach problems, hepatic impairment, mucositis or stomatitis, HFS, pneumonitis, and mode of administration. To reduce severe fatigue to mild-to-moderate fatigue, patients on average would be willing to forego 4.4 months of PFS. To reduce hepatic impairment risk from 0.5% to 0.0%, patients on average would be willing to forego 1.0 month of PFS. The main study limitation was that patients answered hypothetical treatment-choice questions.

CONCLUSIONS

This study provides information to physicians about patient priorities when reviewing and selecting RCC therapies with patients.

摘要

背景

本研究旨在量化并对比二线晚期肾细胞癌(RCC)药物治疗方案及其相关获益和毒性的患者偏好,并帮助构建关于选择合适 RCC 治疗方法的医患讨论。

研究设计和方法

美国成年 RCC 患者完成了一项基于网络的选择格式联合调查,该调查由一系列 10 个治疗选择问题组成,每个问题都包括一对假设的 RCC 药物治疗方案。每个方案都通过各种药物属性(特征或结果)来描述,这些属性具有不同的水平。属性包括疗效(无进展生存期[PFS])、耐受性(疲劳、胃部问题、黏膜炎或口腔炎、手足综合征[HFS])、严重但罕见的不良事件(肺炎、肝损伤)和给药方式。治疗选择问题基于具有已知统计性质的实验设计。随机参数对数回归用于估计每个属性水平的相对偏好权重。还计算了获益等效度量(为毒性获益交换的额外 PFS 月数)。

结果

在完成调查的 272 名患者中,大多数为女性(53%)、白人(92%)且至少拥有大学学历(66%)。平均年龄为 57 岁(标准差:10 岁)。在调查中包含的属性和属性水平范围内,PFS 是最重要的属性,其次是疲劳、胃部问题、肝损伤、黏膜炎或口腔炎、HFS、肺炎和给药方式。为了将严重疲劳减轻至轻度至中度疲劳,患者平均愿意放弃 4.4 个月的 PFS。为了将肝损伤风险从 0.5%降低至 0.0%,患者平均愿意放弃 1.0 个月的 PFS。本研究的主要局限性在于患者回答了假设的治疗选择问题。

结论

本研究为医生在与患者一起审查和选择 RCC 治疗方法时提供了有关患者优先事项的信息。

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