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多发性硬化症疾病修正疗法属性的患者偏好:基于评分的联合分析的开发与结果

Patient preferences for attributes of multiple sclerosis disease-modifying therapies: development and results of a ratings-based conjoint analysis.

作者信息

Wilson Leslie S, Loucks Aimee, Gipson Gregory, Zhong Lixian, Bui Christine, Miller Elizabeth, Owen Mary, Pelletier Daniel, Goodin Douglas, Waubant Emmanuelle, McCulloch Charles E

机构信息

Departments of Clinical Pharmacy (LSW, GG, LZ, CB, EM), Neurology (MO, DG, EW), and Epidemiology and Biostatistics (CEM), University of California, San Francisco, San Francisco, CA, USA; Kaiser Permanente Drug Information Services, Kaiser Permanente, Oakland, CA, USA (AL); and Department of Diagnostic Radiology and Neurology, Yale University School of Medicine, New Haven, CT, USA (DP).

出版信息

Int J MS Care. 2015 Mar-Apr;17(2):74-82. doi: 10.7224/1537-2073.2013-053.

Abstract

BACKGROUND

Timely individualized treatment is essential to improving relapsing-remitting multiple sclerosis (RRMS) patient health outcomes, yet little is known about how patients make treatment decisions. We sought to evaluate RRMS patient preferences for risks and benefits of treatment.

METHODS

Fifty patients with RRMS completed conjoint analysis surveys with 16 hypothetical disease-modifying therapy (DMT) medication profiles developed using a fractional factorial design. Medication profiles were assigned preference ratings from 0 (not acceptable) to 10 (most favorable). Medication attributes included a range of benefits, adverse effects, administration routes, and market durations. Analytical models used linear mixed-effects regression.

RESULTS

Participants showed the highest preference for medication profiles that would improve their symptoms (β = 0.81-1.03, P < .001), not a proven DMT outcome. Preventing relapses, the main clinical trial outcome, was not associated with significant preferences (P = .35). Each year of preventing magnetic resonance imaging changes and disease symptom progression showed DMT preferences of 0.17 point (β = 0.17, P = .002) and 0.12 point (β = 0.12, P < .001), respectively. Daily oral administration was preferred over all parenteral routes (P < .001). A 1% increase in death or severe disability decreased relative DMT preference by 1.15 points (P < .001).

CONCLUSIONS

Patient preference focused on symptoms and prevention of progression but not on relapse prevention, the proven drug outcome. Patients were willing to accept some level of serious risk for certain types and amounts of benefits, and they strongly preferred daily oral administration over all other options.

摘要

背景

及时的个体化治疗对于改善复发缓解型多发性硬化症(RRMS)患者的健康结局至关重要,但对于患者如何做出治疗决策却知之甚少。我们试图评估RRMS患者对治疗风险和获益的偏好。

方法

50例RRMS患者完成了联合分析调查,该调查采用分数析因设计制定了16种假设的疾病修饰治疗(DMT)药物概况。药物概况的偏好评分从0(不可接受)到10(最有利)。药物属性包括一系列获益、不良反应、给药途径和上市时长。分析模型采用线性混合效应回归。

结果

参与者对能够改善其症状的药物概况表现出最高偏好(β = 0.81 - 1.03,P <.001),而这并非DMT已证实的结局。预防复发这一主要临床试验结局与显著偏好无关(P =.35)。预防磁共振成像变化和疾病症状进展的每一年分别显示DMT偏好为0.17分(β = 0.17,P =.002)和0.12分(β = 0.12,P <.001)。每日口服给药比所有胃肠外给药途径更受青睐(P <.001)。死亡或严重残疾增加1%会使DMT相对偏好降低1.15分(P <.001)。

结论

患者偏好集中在症状和疾病进展的预防上,而非已证实的药物结局——预防复发。患者愿意为特定类型和数量的获益接受一定程度的严重风险,并且他们强烈倾向于每日口服给药而非所有其他选择。

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