A Novel Approach to Computing Preference Estimates for Different Treatment Pathways: An Application in Oncology.

IF 3.4 3区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Patient-Patient Centered Outcomes Research Pub Date : 2024-07-01 Epub Date: 2024-03-15 DOI:10.1007/s40271-024-00680-z
Kathleen Beusterien, Oliver Will, Emuella Flood, Susan McCutcheon, deMauri S Mackie, Stella Mokiou
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Abstract

Background: Patients with cancer may progress through multiple treatments with differing adverse effect profiles. Moreover, pathways may be fixed or flexible in allowing for escalation or de-escalation of treatment depending on interim outcomes. We sought to develop a methodology capable of estimating preferences for the entirety of a pathway involving a sequence of different treatments.

Methods: Patients with early breast cancer completed an online discrete choice experiment to assess preferences for eight key early breast cancer attributes. Hierarchical Bayesian modeling was used to calculate attribute-level preference weights. Preference weights for hypothetical pathways were estimated by summing the respective weights for efficacy, flexible or fixed pathway, duration, administration regimen, and adverse event risk, the last two of which were time-adjusted by multiplying each weight by the proportion of time spent on a selected treatment.

Results: Increases in the risk of a serious adverse event were most influential in treatment pathway preferences, followed by increases in efficacy and decreases in overall pathway duration. Patients preferred a flexible pathway versus a fixed pathway. Pathway preference estimates fluctuated in a logically consistent manner. Switching from a flexible to a fixed pathway yielded a significantly lower pathway preference. For this same pathway, when adjuvant treatment was replaced with a treatment with a more favorable toxicity profile and shorter duration, it offset the negative impact of the more toxic neoadjuvant chemotherapy.

Conclusions: This novel methodology accounts for patient preference throughout a sequence of treatments, allowing for comparison of preferences across complex treatment pathways.

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计算不同治疗路径偏好估计值的新方法:肿瘤学应用
背景癌症患者可能会接受多种治疗,但不良反应各不相同。此外,治疗路径可能是固定的,也可能是灵活的,允许根据中期结果升级或降级治疗。我们试图开发一种方法,能够估算出患者对涉及一系列不同治疗的整个治疗路径的偏好:方法:早期乳腺癌患者完成了一项在线离散选择实验,以评估对八种早期乳腺癌关键属性的偏好。使用层次贝叶斯模型计算属性级偏好权重。假设路径的偏好权重是通过将疗效、灵活或固定路径、疗程、给药方案和不良事件风险的各自权重相加来估算的,其中后两个权重是通过将每个权重乘以用于选定治疗的时间比例来进行时间调整的:结果:严重不良事件风险的增加对治疗路径偏好的影响最大,其次是疗效的增加和整个治疗路径持续时间的缩短。与固定治疗路径相比,患者更倾向于灵活的治疗路径。路径偏好估计值的波动在逻辑上是一致的。从灵活路径转为固定路径时,患者对路径的偏好明显降低。对于同一路径,当辅助治疗换成毒性更强、疗程更短的治疗方法时,可以抵消毒性更强的新辅助化疗的负面影响:这种新颖的方法考虑到了患者在整个治疗序列中的偏好,可对复杂治疗路径中的偏好进行比较。
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来源期刊
Patient-Patient Centered Outcomes Research
Patient-Patient Centered Outcomes Research HEALTH CARE SCIENCES & SERVICES-
CiteScore
6.60
自引率
8.30%
发文量
44
审稿时长
>12 weeks
期刊介绍: The Patient provides a venue for scientifically rigorous, timely, and relevant research to promote the development, evaluation and implementation of therapies, technologies, and innovations that will enhance the patient experience. It is an international forum for research that advances and/or applies qualitative or quantitative methods to promote the generation, synthesis, or interpretation of evidence. The journal has specific interest in receiving original research, reviews and commentaries related to qualitative and mixed methods research, stated-preference methods, patient reported outcomes, and shared decision making. Advances in regulatory science, patient-focused drug development, patient-centered benefit-risk and health technology assessment will also be considered. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in The Patient may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances. All manuscripts are subject to peer review by international experts.
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