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肿瘤治疗中离散选择实验的系统评价。

A Systematic Review of Discrete Choice Experiments in Oncology Treatments.

机构信息

Evidera, The Ark, 2nd Floor, 201 Talgarth Road, London, W6 8BJ, UK.

Erasmus University, Rotterdam, The Netherlands.

出版信息

Patient. 2021 Nov;14(6):775-790. doi: 10.1007/s40271-021-00520-4. Epub 2021 May 5.

Abstract

BACKGROUND

As the number and type of cancer treatments available rises and patients live with the consequences of their disease and treatments for longer, understanding preferences for cancer care can help inform decisions about optimal treatment development, access, and care provision. Discrete choice experiments (DCEs) are commonly used as a tool to elicit stakeholder preferences; however, their implementation in oncology may be challenging if burdensome trade-offs (e.g. length of life versus quality of life) are involved and/or target populations are small.

OBJECTIVES

The aim of this review was to characterise DCEs relating to cancer treatments that were conducted between 1990 and March 2020.

DATA SOURCES

EMBASE, MEDLINE, and the Cochrane Database of Systematic Reviews were searched for relevant studies.

STUDY ELIGIBILITY CRITERIA

Studies were included if they implemented a DCE and reported outcomes of interest (i.e. quantitative outputs on participants' preferences for cancer treatments), but were excluded if they were not focused on pharmacological, radiological or surgical treatments (e.g. cancer screening or counselling services), were non-English, or were a secondary analysis of an included study.

ANALYSIS METHODS

Analysis followed a narrative synthesis, and quantitative data were summarised using descriptive statistics, including rankings of attribute importance.

RESULT

Seventy-nine studies were included in the review. The number of published DCEs relating to oncology grew over the review period. Studies were conducted in a range of indications (n = 19), most commonly breast (n =10, 13%) and prostate (n = 9, 11%) cancer, and most studies elicited preferences of patients (n = 59, 75%). Across reviewed studies, survival attributes were commonly ranked as most important, with overall survival (OS) and progression-free survival (PFS) ranked most important in 58% and 28% of models, respectively. Preferences varied between stakeholder groups, with patients and clinicians placing greater importance on survival outcomes, and general population samples valuing health-related quality of life (HRQoL). Despite the emphasis of guidelines on the importance of using qualitative research to inform attribute selection and DCE designs, reporting on instrument development was mixed.

LIMITATIONS

No formal assessment of bias was conducted, with the scope of the paper instead providing a descriptive characterisation. The review only included DCEs relating to cancer treatments, and no insight is provided into other health technologies such as cancer screening. Only DCEs were included.

CONCLUSIONS AND IMPLICATIONS

Although there was variation in attribute importance between responder types, survival attributes were consistently ranked as important by both patients and clinicians. Observed challenges included the risk of attribute dominance for survival outcomes, limited sample sizes in some indications, and a lack of reporting about instrument development processes.

PROTOCOL REGISTRATION

PROSPERO 2020 CRD42020184232.

摘要

背景

随着癌症治疗方法的数量和类型的增加,以及患者需要长期应对疾病和治疗的后果,了解癌症护理偏好可以帮助我们做出有关最佳治疗开发、可及性和护理提供的决策。离散选择实验(DCE)通常被用作获取利益相关者偏好的工具;然而,如果涉及到令人为难的权衡(例如,寿命与生活质量),或者目标人群较小,那么在肿瘤学中实施 DCE 可能具有挑战性。

目的

本综述的目的是描述 1990 年至 2020 年 3 月期间进行的与癌症治疗相关的 DCE。

数据来源

EMBASE、MEDLINE 和 Cochrane 系统评价数据库被用于搜索相关研究。

研究入选标准

如果实施了 DCE 并报告了感兴趣的结果(即参与者对癌症治疗偏好的定量结果),则纳入研究,但如果研究不专注于药物、放射或手术治疗(例如癌症筛查或咨询服务)、非英文或为纳入研究的二次分析,则排除研究。

分析方法

分析遵循叙述性综合,使用描述性统计数据(包括属性重要性排名)总结定量数据。

结果

该综述共纳入 79 项研究。与肿瘤学相关的 DCE 出版物数量在综述期间有所增加。研究在多种适应症中进行(n=19),最常见的是乳腺癌(n=10,13%)和前列腺癌(n=9,11%),大多数研究都评估了患者的偏好(n=59,75%)。在综述研究中,生存属性通常被列为最重要的属性,总生存期(OS)和无进展生存期(PFS)分别在 58%和 28%的模型中被列为最重要的属性。利益相关者群体之间的偏好存在差异,患者和临床医生更看重生存结果,而一般人群样本则看重健康相关生活质量(HRQoL)。尽管指南强调使用定性研究来为属性选择和 DCE 设计提供信息的重要性,但仪器开发报告存在差异。

局限性

未进行正式的偏倚评估,本文的范围仅提供了描述性特征。该综述仅包括与癌症治疗相关的 DCE,并未提供其他卫生技术(如癌症筛查)的相关信息。仅纳入了 DCE。

结论和意义

尽管应答者类型之间的属性重要性存在差异,但生存属性始终被患者和临床医生列为重要属性。观察到的挑战包括生存结果属性的主导风险、某些适应症中的样本量有限,以及缺乏有关仪器开发过程的报告。

注册协议

PROSPERO 2020 CRD42020184232。

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