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采用多维展开法评估多发性硬化症患者对疾病修饰治疗的偏好:一项试点研究。

Using a multidimensional unfolding approach to assess multiple sclerosis patient preferences for disease-modifying therapy: a pilot study.

作者信息

Sempere Angel Perez, Vera-Lopez Vanesa, Gimenez-Martinez Juana, Ruiz-Beato Elena, Cuervo Jesús, Maurino Jorge

机构信息

Department of Neurology, Hospital General Universitario de Alicante, Alicante.

Department of Statistics, University of Salamanca, Salamanca.

出版信息

Patient Prefer Adherence. 2017 May 29;11:995-999. doi: 10.2147/PPA.S129356. eCollection 2017.

Abstract

PURPOSE

Multidimensional unfolding is a multivariate method to assess preferences using a small sample size, a geometric model locating individuals and alternatives as points in a joint space. The objective was to evaluate relapsing-remitting multiple sclerosis (RRMS) patient preferences toward key disease-modifying therapy (DMT) attributes using multidimensional unfolding.

PATIENTS AND METHODS

A cross-sectional pilot study in RRMS patients was conducted. Drug attributes included relapse prevention, disease progression prevention, side-effect risk and route and schedule of administration. Assessment of preferences was performed through a five-card game. Patients were asked to value attributes from 1 (most preferred) to 5 (least preferred).

RESULTS

A total of 37 patients were included; the mean age was 38.6 years, and 78.4% were female. Disease progression prevention was the most important factor (51.4%), followed by relapse prevention (40.5%). The frequency of administration had the lowest preference rating for 56.8% of patients. Finally, 19.6% valued the side-effect risk attribute as having low/very low importance.

CONCLUSION

Patients' perspective for DMT attributes may provide valuable information to facilitate shared decision-making. Efficacy attributes were the most important drug characteristics for RRMS patients. Multidimensional unfolding seems to be a feasible approach to assess preferences in multiple sclerosis patients. Further elicitation studies using multidimensional unfolding with other stated choice methods are necessary to confirm these findings.

摘要

目的

多维展开是一种使用小样本量评估偏好的多变量方法,是一种将个体和备选方案定位为联合空间中点的几何模型。目的是使用多维展开来评估复发缓解型多发性硬化症(RRMS)患者对关键疾病修正疗法(DMT)属性的偏好。

患者与方法

对RRMS患者进行了一项横断面试点研究。药物属性包括预防复发、预防疾病进展、副作用风险以及给药途径和时间表。通过五张牌游戏进行偏好评估。要求患者对属性从1(最偏好)到5(最不偏好)进行评分。

结果

共纳入37例患者;平均年龄为38.6岁,78.4%为女性。预防疾病进展是最重要的因素(51.4%),其次是预防复发(40.5%)。56.8%的患者对给药频率的偏好评分最低。最后,19.6%的患者认为副作用风险属性的重要性较低/非常低。

结论

患者对DMT属性的看法可能为促进共同决策提供有价值的信息。疗效属性是RRMS患者最重要的药物特征。多维展开似乎是评估多发性硬化症患者偏好的一种可行方法。有必要使用多维展开与其他陈述性选择方法进行进一步的诱导研究以证实这些发现。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9174/5460668/47d397bc012b/ppa-11-995Fig1.jpg

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