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转移性结直肠癌治疗的患者偏好:一种使用离散选择实验和最佳-最差尺度法的多方法途径

Patient Preferences for Metastatic Colorectal Cancer Treatment: A Multi-method Approach Using Discrete Choice Experiments and Best-Worst Scaling.

作者信息

Oedingen Carina, MacDonald Karen V, Stein Barry D, Batist Gerald, Cheung Winson Y, Gill Sharlene, Goldenberg Benjamin A, Ko Yoo-Joung, Marshall Deborah A

机构信息

Department of Community Health Sciences, Cumming School of Medicine, Health Research Innovation Centre (HRIC) - 3C56, University of Calgary, 3280 Hospital Drive NW, Calgary, Alberta T2N 4Z6, Canada.

Colorectal Cancer Canada, Montréal, QC, Canada.

出版信息

Patient. 2025 Jul 29. doi: 10.1007/s40271-025-00760-8.

Abstract

BACKGROUND

Treatment decisions for metastatic colorectal cancer (mCRC) require patients to balance survival benefits, health-related quality of life (HRQoL), and potential risks of side effects while also factoring in their own preferences for different treatment options. Despite growing interest, quantitative patient preferences are not yet integrated into health technology assessments (HTAs) for drug reimbursement recommendations.

OBJECTIVES

The Colorectal Cancer Canada's Patient Values Project aims to explore approaches to incorporate quantitative patient preferences into cancer treatment HTA decision-making processes. As a first step, we elicited the treatment preferences and risk tolerance of patients with mCRC in Canada using a multi-method approach.

METHODS

We developed a preference survey that included two discrete choice experiments (DCEs) and case 1 best-worst scaling (BWS-1) to estimate preferences for mCRC treatments. DCE1 included change in HRQoL and overall survival as attributes, and treatment attributes in DCE2 were administration and frequency, side effects (nausea, pain, diarrhea), and progression-free survival. The BWS-1 included 25 attributes of potential mCRC treatment side effects based on the cancer-specific quality-of-life questionnaire. The survey was administered across Canada to patients with mCRC aged ≥ 18 years with a self-reported diagnosis of mCRC through patient organizations, cancer centers, and an online panel. Data were analyzed using mixed logit and latent class models (DCEs) and count-based analysis (BWS-1).

RESULTS

Overall, 127 patients with mCRC completed the full survey (n = 143 fully completed DCE1, n = 108 fully completed DCE2, n = 127 fully completed BWS-1). Relative preferences for the treatment attributes in the study were consistent with the expectation that better clinical outcomes were preferred over worse clinical outcomes. In DCE1, patients valued both overall survival (24 vs. 12 months) and HRQoL (improvement to 90 vs. worsens to 50 out of 100) as almost equally important. In DCE2, patients preferred better outcomes (longer progression-free survival and no side effects) over worse outcomes, with a disutility for oral capsules/pills compared with intravenous infusions. Significant preference heterogeneity was observed depending on experiences with CRC treatments, treatment side effects, and health status. In the BWS-1, "need help with eating, dressing, washing yourself or using the toilet", "vomiting", and "pain" were ranked as the least and "need to rest", "trouble doing strenuous activities", and "feel tired" as the most tolerable side effects.

CONCLUSIONS

This study highlights the value of a multi-method approach in comprehensively assessing treatment preferences and risk tolerance in mCRC. By triangulating multiple preference-elicitation methods, our findings offer a more robust foundation for integrating patient perspectives into Canada's HTA framework. These results will inform the next step of the Colorectal Cancer Canada's Patient Values Project, which aims to explore approaches to explicitly incorporate patient preferences alongside clinical and economic evidence into the cancer treatment HTA decision-making process in Canada.

摘要

背景

转移性结直肠癌(mCRC)的治疗决策要求患者在生存获益、健康相关生活质量(HRQoL)和潜在副作用风险之间进行权衡,同时还要考虑自身对不同治疗方案的偏好。尽管关注度不断提高,但定量的患者偏好尚未纳入用于药物报销建议的卫生技术评估(HTA)中。

目的

加拿大结直肠癌患者价值观项目旨在探索将定量的患者偏好纳入癌症治疗HTA决策过程的方法。作为第一步,我们采用多方法对加拿大mCRC患者的治疗偏好和风险承受能力进行了调查。

方法

我们开发了一项偏好调查,其中包括两个离散选择实验(DCE)和案例1最佳-最差尺度法(BWS-1),以评估对mCRC治疗的偏好。DCE1将HRQoL的变化和总生存期作为属性,DCE2中的治疗属性为给药方式和频率、副作用(恶心、疼痛、腹泻)以及无进展生存期。BWS-1基于癌症特异性生活质量问卷,纳入了25种mCRC潜在治疗副作用的属性。该调查通过患者组织、癌症中心和在线小组在加拿大各地对年龄≥18岁、自我报告诊断为mCRC的患者进行。使用混合逻辑模型和潜在类别模型(DCE)以及基于计数的分析(BWS-1)对数据进行分析。

结果

总体而言,127例mCRC患者完成了完整调查(n = 143例完全完成DCE1,n = 108例完全完成DCE2,n = 127例完全完成BWS-1)。研究中对治疗属性的相对偏好与以下预期一致,即较好的临床结果优于较差的临床结果。在DCE1中,患者认为总生存期(24个月与12个月)和HRQoL(从100分中的50分改善到90分)几乎同样重要。在DCE2中,患者更喜欢较好的结果(更长的无进展生存期和无副作用)而非较差的结果,与静脉输注相比,口服胶囊/片剂的效用较低。根据结直肠癌治疗经验、治疗副作用和健康状况观察到显著的偏好异质性。在BWS-1中,“吃饭、穿衣、洗漱或上厕所需要帮助”“呕吐”和“疼痛”被列为最难以耐受的副作用,而“需要休息”“进行剧烈活动有困难”和“感到疲倦”被列为最能耐受的副作用。

结论

本研究强调了多方法在全面评估mCRC治疗偏好和风险承受能力方面的价值。通过对多种偏好诱导方法进行三角测量,我们的研究结果为将患者观点纳入加拿大HTA框架提供了更坚实的基础。这些结果将为加拿大结直肠癌患者价值观项目的下一步工作提供信息,该项目旨在探索将患者偏好与临床和经济证据明确纳入加拿大癌症治疗HTA决策过程的方法。

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