Department of Epidemiology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
European Medicines Agency, London, United Kingdom.
Oncologist. 2018 Jan;23(1):44-51. doi: 10.1634/theoncologist.2017-0257. Epub 2017 Oct 27.
The objectives of this study were to elicit the preferences of patients with multiple myeloma regarding the possible benefits and risks of cancer treatments and to illustrate how such data may be used to estimate patients' acceptance of new treatments.
Patients with multiple myeloma from the cancer charity Myeloma UK were invited to participate in an online survey based on multicriteria decision analysis and swing weighting to elicit individual stated preferences for the following attributes: (a) 1-year progression-free survival (PFS, ranging from 50% to 90%), (b) mild or moderate toxicity for 2 months or longer (ranging from 85% to 45%), and (c) severe or life-threatening toxicity (ranging from 80% to 20%).
A total of 560 participants completed the survey. The average weight given to PFS was 0.54, followed by 0.32 for severe or life-threatening toxicity and 0.14 for mild or moderate chronic toxicity. Participants who ranked severe or life-threatening toxicity above mild or moderate chronic toxicity (56%) were more frequently younger, working, and looking after dependent family members and had more frequently experienced severe or life-threatening side effects. The amount of weight given to PFS did not depend on any of the collected covariates. The feasibility of using the collected preference data to estimate the patients' acceptance of specific multiple myeloma treatments was demonstrated in a subsequent decision analysis example.
Stated preference studies provide a systematic approach to gain knowledge about the distribution of preferences in the population and about what this implies for patients' acceptance of specific treatments.
This study demonstrated how quantitative preference statements from a large group of participants can be collected through an online survey and how such information may be used to explore the acceptability of specific treatments based on the attributes studied. Results from such studies have the potential to become an important new tool for gathering patient views and studying heterogeneity in preferences in a systematic way, along with other methods, such as focus groups and expert opinions.
本研究旨在了解多发性骨髓瘤患者对癌症治疗可能获益和风险的偏好，并举例说明了如何利用这些数据来评估患者对新疗法的接受程度。
英国多发性骨髓瘤慈善机构 Myeloma UK 的多发性骨髓瘤患者受邀参与了一项基于多准则决策分析和摇摆权重的在线调查，以了解患者对以下属性的个人偏好：（a）1 年无进展生存期（PFS，范围为 50%至 90%），（b）2 个月或更长时间的轻度或中度毒性（范围为 85%至 45%），和（c）严重或危及生命的毒性（范围为 80%至 20%）。
共有 560 名参与者完成了调查。PFS 的平均权重为 0.54，其次是严重或危及生命的毒性为 0.32，轻度或中度慢性毒性为 0.14。将严重或危及生命的毒性排在轻度或中度慢性毒性之上的参与者（56%）更年轻、在职且照顾需要依赖他人的家庭成员，并且更频繁地经历严重或危及生命的副作用。给予 PFS 的权重与收集到的任何协变量无关。在随后的决策分析示例中，证明了使用收集到的偏好数据来估计患者对特定多发性骨髓瘤治疗方法的接受程度的可行性。
基于意愿的研究提供了一种系统的方法来了解人群中偏好的分布情况，以及这对患者对特定治疗方法的接受程度意味着什么。
本研究展示了如何通过在线调查收集大量参与者的定量偏好陈述，以及如何利用这些信息根据所研究的属性来探索特定治疗方法的可接受性。此类研究的结果有可能成为一种重要的新工具，用于系统地收集患者意见并研究偏好的异质性，与其他方法（如焦点小组和专家意见）一起使用。
