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患者在选择慢性淋巴细胞白血病治疗方法时的优先考虑因素。

Patients' priorities in selecting chronic lymphocytic leukemia treatments.

作者信息

Mansfield Carol, Masaquel Anthony, Sutphin Jessie, Weiss Elisa, Gutierrez Meghan, Wilson Jennifer, Boeri Marco, Li Jia, Reyes Carolina

机构信息

RTI Health Solutions, Research Triangle Park, NC.

Genentech Inc., South San Francisco, CA.

出版信息

Blood Adv. 2017 Oct 31;1(24):2176-2185. doi: 10.1182/bloodadvances.2017007294. eCollection 2017 Nov 14.

Abstract

Currently, in the United States, 130 000 people live with chronic lymphocytic leukemia (CLL), and almost 20 000 new cases of CLL are diagnosed each year. Little is known about the value patients place upon the attributes of available CLL treatments, which vary in efficacy, side effects, and mode of administration. We used a discrete-choice experiment (DCE) to investigate patients' preferences for treatment attributes and the impact of out-of-pocket cost on patients' choices. DCE surveys pose a series of choices between hypothetical treatment options, each defined by a set of attributes, and the responses provide quantitative estimates of the average relative preferences for treatment attribute. Each hypothetical treatment in this survey was defined by 5 attributes with predefined levels for efficacy, adverse events, and mode administration. A patient advocacy organization recruited 384 patients with a self-reported physician diagnosis of CLL to complete the online survey. Respondents placed the highest relative importance on longer progression-free survival (PFS). However, the risk of adverse events also was important, as significant additional PFS was needed to offset patients' acceptance of worsening adverse events. A supplemental question with 2 treatments and varying costs was included to assess the impact of cost on choice. When costs were included, a large proportion of patients changed their choices between the 2 treatments. Given the available treatments and the high cost of some treatments, physicians may want to explore their patients' preferences for different treatment features, including benefit-risk tradeoffs and out-of-pocket cost, when selecting the best treatment strategies for patients.

摘要

目前,在美国,有13万人患有慢性淋巴细胞白血病(CLL),每年新增近2万例CLL病例。对于患者如何看待现有CLL治疗方法的属性,人们知之甚少,这些治疗方法在疗效、副作用和给药方式上各不相同。我们采用离散选择实验(DCE)来研究患者对治疗属性的偏好以及自付费用对患者选择的影响。DCE调查在一系列假设的治疗方案之间进行选择,每个方案由一组属性定义,回答提供了对治疗属性平均相对偏好的定量估计。本次调查中的每个假设治疗方案由5个属性定义,这些属性在疗效、不良事件和给药方式方面有预先设定的水平。一个患者权益倡导组织招募了384名自我报告经医生诊断为CLL的患者来完成在线调查。受访者对更长的无进展生存期(PFS)赋予了最高的相对重要性。然而,不良事件的风险也很重要,因为需要显著延长PFS才能抵消患者对不良事件恶化的接受程度。包含了一个关于两种治疗方法及不同费用的补充问题,以评估费用对选择的影响。当考虑费用时,很大一部分患者改变了他们在两种治疗方法之间的选择。鉴于现有的治疗方法以及某些治疗方法的高成本,医生在为患者选择最佳治疗策略时,可能需要探究患者对不同治疗特征的偏好,包括获益-风险权衡和自付费用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/efa3/5737119/885da7ea16e4/advances007294absf1.jpg

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