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患者偏好确实很重要:一项在六个欧洲国家进行的针对乳腺癌患者的离散选择实验,采用潜在类别分析。

Patient preferences do matter: a discrete choice experiment conducted with breast cancer patients in six European countries, with latent class analysis.

机构信息

Pharmecons Easy Access Ltd, York, UK.

Novartis Oncology Region Europe, Origgio, Italy.

出版信息

Int J Technol Assess Health Care. 2023 Apr 19;39(1):e21. doi: 10.1017/S0266462323000168.

Abstract

OBJECTIVES

The evolution of breast cancer (BC) treatments has resulted in tailored therapies for the different types and stages of BC. Each treatment has a profile of benefits and adverse effects which are taken into consideration when planning a treatment pathway. This study examines whether patients' preferences are in line with what is considered important from decision makers viewpoint.

METHODS

An online discrete choice experiment was conducted in six European countries (France, Germany, Ireland, Poland, Spain, UK) with BC patients. Six attributes were included: overall survival (OS), hyperglycemia, rash, pain, functional well-being (FWB), and out-of-pocket payment (OOP). Sixteen choice sets with two hypothetical treatments and a "No treatment" option were presented. Data were analyzed with the use of heteroscedastic conditional, mixed logistic, and latent class models. Marginal rate of substitution (MRS) were estimated for OOP versus the rest of attributes to establish the ranking of preferences for each attribute.

RESULTS

Two hundred and forty-seven patients with advanced or metastatic BC and 314 with early-stage BC responded. Forty-nine percent of patients were < 44 years old and 65 percent had completed university education. The MRS of the analysis demonstrated that "severe pain" is the highest dis-preferred attribute level, followed by "severe impairment in FWB" and OS. Four classes of patients as "decision makers" were identified.

CONCLUSIONS

This study suggests that there is heterogeneity in treatment preferences of BC patients depending on their sociodemographic and disease-related characteristics. In combination with clinical guidelines, patient preferences can support the selection and tailoring of treatment options.

摘要

目的

乳腺癌(BC)治疗方法的发展导致了针对不同类型和阶段 BC 的定制疗法。每种治疗方法都有其获益和不良反应的特征,在制定治疗方案时会考虑到这些特征。本研究考察了患者的偏好是否与决策者认为重要的因素一致。

方法

在六个欧洲国家(法国、德国、爱尔兰、波兰、西班牙和英国)对 BC 患者进行了在线离散选择实验。纳入了 6 个属性:总生存期(OS)、高血糖、皮疹、疼痛、功能健康状况(FWB)和自付费用(OOP)。共呈现了 16 个选择集,其中包括两种假设治疗方案和“无治疗”选项。使用异方差条件混合逻辑和潜在类别模型对数据进行了分析。对 OOP 与其他属性进行了边际替代率(MRS)的估计,以确定每个属性的偏好排序。

结果

共有 247 名晚期或转移性 BC 患者和 314 名早期 BC 患者做出了回应。49%的患者年龄<44 岁,65%的患者完成了大学教育。分析的 MRS 表明,“严重疼痛”是最不受欢迎的属性水平,其次是“FWB 严重受损”和 OS。确定了 4 类“决策者”患者。

结论

本研究表明,BC 患者的治疗偏好存在异质性,这取决于他们的社会人口统计学和疾病相关特征。结合临床指南,患者的偏好可以支持治疗选择的选择和定制。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f8b1/11574541/89bdf9f0308b/S0266462323000168_fig1.jpg

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