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肺癌患者偏好分析——评估治疗益处与副作用之间可接受的权衡

Analysis of Patient Preferences in Lung Cancer - Estimating Acceptable Tradeoffs Between Treatment Benefit and Side Effects.

作者信息

Janssen Ellen M, Dy Sydney M, Meara Alexa S, Kneuertz Peter J, Presley Carolyn J, Bridges John F P

机构信息

Center for Medical Technology Policy, Baltimore, MD, USA.

Department of Health Policy and Management, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD, USA.

出版信息

Patient Prefer Adherence. 2020 Jun 3;14:927-937. doi: 10.2147/PPA.S235430. eCollection 2020.

Abstract

OBJECTIVE

Increased treatment options and longer survival for lung cancer have generated increased interest in patient preferences. Previous studies of patient preferences in lung cancer have not fully explored preference heterogeneity. We demonstrate a method to explore preference heterogeneity in the willingness of patients with lung cancer and caregivers to trade progression-free survival (PFS) with side effects.

PATIENTS AND METHODS

Patients and caregivers attending a national lung cancer meeting completed a discrete-choice experiment (DCE) designed through a collaboration with patients. Participants answered 13 choice tasks described across PFS, short-term side effects, and four long-term side effects. Side effects were coded as a one-level change in severity (none-mild, mild-moderate, or moderate-severe). A mixed logit model in willingness-to-pay space estimated preference heterogeneity in acceptable tradeoffs (time equivalents) between PFS and side effects. The study was reported following quality indicators from the United States Food and Drug Administration's patient preference guidance.

RESULTS

A total of 87 patients and 24 caregivers participated in the DCE. Participants would trade 3.7 month PFS (95% CI (CI): 3.3-4.1) for less severe functional long-term treatment side effects, 2.3 months for less severe physical long-term effects (CI: 1.9-2.8) and cognitive long-term effects (CI: 1.8-2.8), 0.9 months (CI: 0.4-1.4) for less severe emotional long-term effects, and 1.8 months (CI: 1.4-2.3) for less severe short-term side effects. Most participants (90%) would accept treatment with more severe functional long-term effects for 8.4 additional month PFS.

CONCLUSION

Participants would trade PFS for changes in short-term side effects and long-term side effects, although preference heterogeneity existed. Lung cancer treatments that offer less PFS but also less severe side effects might be acceptable to some patients.

摘要

目的

肺癌治疗方案的增加以及患者生存期的延长使得人们对患者偏好的关注度日益提高。以往关于肺癌患者偏好的研究尚未充分探讨偏好的异质性。我们展示了一种方法,用于探究肺癌患者及其护理人员在无进展生存期(PFS)与副作用之间进行权衡的意愿中的偏好异质性。

患者与方法

参加全国肺癌会议的患者及其护理人员完成了一项通过与患者合作设计的离散选择实验(DCE)。参与者回答了13项关于PFS、短期副作用和四项长期副作用的选择任务。副作用被编码为严重程度的一级变化(无-轻度、轻度-中度或中度-重度)。在支付意愿空间中的混合逻辑模型估计了PFS与副作用之间可接受权衡(时间等价物)中的偏好异质性。该研究按照美国食品药品监督管理局患者偏好指南的质量指标进行报告。

结果

共有87名患者和24名护理人员参与了DCE。参与者愿意用3.7个月的PFS(95%置信区间(CI):3.3 - 4.1)来换取不太严重的长期功能治疗副作用,用2.3个月换取不太严重的长期身体副作用(CI:1.9 - 2.8)和认知长期副作用(CI:1.8 - 2.8),用0.9个月换取不太严重的长期情绪副作用(CI:0.4 - 1.4),用1.8个月换取不太严重的短期副作用。大多数参与者(90%)愿意接受具有更严重长期功能影响的治疗,以换取额外8.4个月的PFS。

结论

尽管存在偏好异质性,但参与者愿意用PFS来换取短期和长期副作用的变化。对于一些患者来说,提供较短PFS但副作用也较轻的肺癌治疗方案可能是可以接受的。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a5d7/7276327/7169380e64a4/PPA-14-927-g0001.jpg

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