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骨髓增生异常综合征患者对低甲基化药物的获益、风险和给药途径的偏好。

Patient Preferences for Benefits, Risks, and Administration Route of Hypomethylating Agents in Myelodysplastic Syndromes.

机构信息

Section of Hematology, Department of Medicine, Yale School of Medicine, and Yale Cancer Center, New Haven, CT.

OPEN Health, Bethesda, MD.

出版信息

Clin Lymphoma Myeloma Leuk. 2022 Sep;22(9):e853-e866. doi: 10.1016/j.clml.2022.04.023. Epub 2022 May 2.

Abstract

INTRODUCTION/BACKGROUND: Therapy with infused or injected hypomethylating agents (HMAs) may lead to higher treatment administration burden (ie, local reaction, visit frequency and duration) vs. oral HMAs.   OBJECTIVES: To reveal preferences of US and Canadian patients with myelodysplastic syndromes (MDS) for HMAs' benefits, risks, and administration burden through an online discrete-choice experiment (DCE).

MATERIALS AND METHODS

Choice of DCE attributes and survey development were informed by literature review and interviews with clinicians, MDS patients, and caregivers serving as patient proxies, and patient advocacy groups (PAGs) representatives, including from AAMAC, AAMDS, and MDSF. DCE choice tasks were analyzed using random parameter logit models. Survey patients were recruited by the PAGs via their networks. To understand key preference drivers and how much patients were willing to trade between attributes, we calculated each attribute's relative attribute importance (RAI) and marginal rates of substitution.

RESULTS

One hundred eighty-four respondents (including 158 patients; mean age, 67.2 years; male, 50.5%; White, 50.5%; US residents, 88%) completed the survey. MDS risk was low (34.8%), high (30.9%), or unknown (34.2%). RAI (in decreasing order) was as follows: risk of AML (40%), fatigue level (33%), number of visits (12%), mode of administration (6%), visit duration (5%), and administration frequency (4%). Assuming the same risk of AML transformation or level of fatigue, most respondents (76.6%) were predicted to switch to an oral pill if it were available to them.

CONCLUSION

Given equivalent effectiveness across HMAs, patients' preferences for HMA administration method should be considered in treatment decision-making to minimize burden and facilitate adherence.

摘要

介绍/背景:与口服低甲基化药物(HMAs)相比,输注或注射 HMAs 可能会导致更高的治疗管理负担(即局部反应、就诊频率和持续时间)。目的:通过在线离散选择实验(DCE)揭示美国和加拿大骨髓增生异常综合征(MDS)患者对 HMA 益处、风险和管理负担的偏好。材料与方法:通过文献回顾和对临床医生、MDS 患者和作为患者代理人的护理人员以及代表患者权益团体(PAG)的访谈,了解 DCE 属性的选择和调查的制定,包括来自 AAMAC、AAMDS 和 MDSF 的 PAG。使用随机参数对数模型分析 DCE 选择任务。通过 PAG 通过他们的网络招募调查患者。为了了解关键偏好驱动因素以及患者在属性之间的意愿交易程度,我们计算了每个属性的相对属性重要性(RAI)和边际替代率。结果:184 名受访者(包括 158 名患者;平均年龄 67.2 岁;男性 50.5%;白人 50.5%;美国居民 88%)完成了调查。MDS 风险低(34.8%)、高(30.9%)或未知(34.2%)。RAI(降序排列)如下:AML 风险(40%)、疲劳程度(33%)、就诊次数(12%)、给药方式(6%)、就诊持续时间(5%)和给药频率(4%)。如果有口服药丸可供使用,大多数受访者(76.6%)预计会在同样的 AML 转化风险或疲劳水平下选择转为口服药丸。结论:在 HMAs 疗效相当的情况下,在治疗决策中应考虑患者对 HMA 给药方式的偏好,以最大程度地减轻负担并提高依从性。

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