Background

There is a growing consensus that the perspective of the patient should be considered in the evaluation of novel interventions.

Research Question

What treatment outcomes matter to people with cystic fibrosis (CF), and what trade-offs would they make to realize these outcomes?

Study Design and Methods

Adults attending a specialist CF center were invited to complete an online discrete choice experiment (DCE). The DCE required participants to evaluate hypothetical CF treatment profiles, defined by impact on lung function, pulmonary exacerbations, abdominal symptoms, life expectancy, quality of life, inhaled medicine usage, and physiotherapy requirement. Choice data were analyzed, using multinomial logit and latent class models.

Results

One hundred and three people with CF completed the survey (median age, 35 years; range, 18-76 years); 52% were female; mean FEV₁ % predicted, 69% [SD, 22%]). On average, an improvement in life expectancy by 10 years or more had the greatest impact on treatment preference, followed by a 15% increase in lung function. However, it was shown that people would trade substantial reductions in these key outcomes to reduce treatment time or burden. Preference profiles were not uniform across the sample: three distinct subgroups were identified, each placing markedly different importance on the relative importance of both life expectancy and lung function compared with other attributes.

Interpretation

The relative importance of treatment burden to people with CF, compared with life expectancy and lung function, suggests it should be routinely captured in clinical trials as an important secondary outcome measure. When considering the patient perspective, it is important that decision-makers recognize that the values of people with CF are not homogeneous.

中文翻译：

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囊性纤维化患者的治疗偏好

背景

人们越来越一致认为，在评估新干预措施时应考虑患者的观点。

研究问题

哪些治疗结果对囊性纤维化 (CF) 患者很重要，他们会做出哪些权衡来实现这些结果？

研究设计和方法

邀请参加专业 CF 中心的成年人完成在线离散选择实验 (DCE)。DCE 要求参与者评估假设的 CF 治疗方案，定义为对肺功能的影响、肺部恶化、腹部症状、预期寿命、生活质量、吸入药物的使用和物理治疗要求。使用多项逻辑和潜在类别模型分析了选择数据。

结果

103 名 CF 患者完成了调查（中位年龄，35 岁；范围，18-76 岁）；52% 为女性；平均 FEV ₁ % 预测值，69% [SD, 22%]）。平均而言，预期寿命提高 10 年或更长时间对治疗偏好的影响最大，其次是肺功能提高 15%。然而，事实表明，人们会以大幅减少这些关键结果来换取减少治疗时间或负担。样本中的偏好配置文件并不统一：确定了三个不同的亚组，每个亚组对预期寿命和肺功能与其他属性的相对重要性的重视程度明显不同。

解释

与预期寿命和肺功能相比，治疗负担对 CF 患者的相对重要性表明，在临床试验中应将其作为重要的次要结果指标定期捕获。在考虑患者的观点时，重要的是决策者要认识到 CF 患者的价值观是不同的。