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I-PreFer研究:一项离散选择实验,以探索患者、护理人员和肺科医生对特发性肺纤维化药物治疗方案的偏好。

I-PreFer Study: A Discrete Choice Experiment to Explore Patient, Caregiver and Pulmonologist Preferences of Idiopathic Pulmonary Fibrosis Pharmacological Treatment Options.

作者信息

Hollmen Maria, Wijsenbeek Marlies, Bromilow Tom, Smith Adam B, Mealing Stuart, Lewis Damian, Galvin Liam, Jones Steve, Asijee Guus, Soulard Stéphane, Froidure Antoine

机构信息

Faculty of Medicine, University of Helsinki, Helsinki, Finland.

Centre of Excellence for Interstitial Lung Diseases and Sarcoidosis, Department of Respiratory Medicine, Erasmus MC, University Medical Center, Rotterdam, the Netherlands.

出版信息

Patient Prefer Adherence. 2023 Aug 4;17:1895-1906. doi: 10.2147/PPA.S409767. eCollection 2023.

Abstract

PURPOSE

Idiopathic pulmonary fibrosis (IPF) is a chronic, progressive, and ultimately fatal lung disease that, while rare, has seen incidence rise over time. There is no cure for IPF other than a lung transplant, though two antifibrotic (AF) drugs do exist to slow disease progression. While these drugs are efficacious, they are both associated with differing profiles of adverse events. This study aimed to elicit patient, caregiver and pulmonologist preferences on the treatment profiles of AFs via a discrete choice experiment (DCE).

PATIENTS AND METHODS

The DCE and associated survey were distributed across 7 European countries, and bespoke DCEs were developed for patients/caregivers and pulmonologists. After collaboration with European Pulmonary Fibrosis & Related Disorders Federation (EU-PFF) and expert pulmonologists, respectively, a patient/caregiver DCE with 5 attributes and a pulmonologist DCE with 6 attributes were finalized. The DCEs had a blocked approach to reduce participant burden and were distributed on an online survey platform. Preferences were estimated through conditional multinomial logit regression analysis.

RESULTS

Ninety-five patients, 22 caregivers and 115 pulmonologists fully completed their respective DCEs. Overall, patients and caregivers preferred management of treatment-related adverse events over both survival benefits and disease progression. Nearly all preference levels were found to be significantly different from their reference level. In contrast, pulmonologists showed a greater preference for control of lung function and exacerbations over adverse events. Although there were relative differences between the univariate subgroups in terms of the preference weights, most of these were not statistically significant.

CONCLUSION

The outcomes from this study suggest that while patients and caregivers had similar preferences for characteristics of IPF treatments, pulmonologists did not share those same preferences. Patients and caregivers preferred safety, while pulmonologists preferred efficacy. These differences should be considered by clinicians to better involve the patient in treatment decision-making for IPF.

摘要

目的

特发性肺纤维化(IPF)是一种慢性、进行性且最终致命的肺部疾病,虽然罕见,但发病率随时间呈上升趋势。除了肺移植外,IPF 无法治愈,不过有两种抗纤维化(AF)药物可减缓疾病进展。虽然这些药物有效,但它们都与不同的不良事件特征相关。本研究旨在通过离散选择实验(DCE)得出患者、护理人员和肺科医生对 AF 治疗特征的偏好。

患者与方法

DCE 及相关调查问卷在 7 个欧洲国家分发,针对患者/护理人员和肺科医生分别开发了定制的 DCE。分别与欧洲肺纤维化及相关疾病联合会(EU-PFF)和肺科专家合作后,最终确定了一个包含 5 个属性的患者/护理人员 DCE 和一个包含 6 个属性的肺科医生 DCE。DCE 采用分块方法以减轻参与者负担,并在在线调查平台上分发。通过条件多项逻辑回归分析估计偏好。

结果

95 名患者、22 名护理人员和 115 名肺科医生完全完成了各自的 DCE。总体而言,患者和护理人员更倾向于管理与治疗相关的不良事件,而非生存获益和疾病进展。几乎所有偏好水平与参考水平均有显著差异。相比之下,肺科医生更倾向于控制肺功能和病情加重,而非不良事件。虽然单变量亚组在偏好权重方面存在相对差异,但大多数差异无统计学意义。

结论

本研究结果表明,虽然患者和护理人员对 IPF 治疗特征有相似偏好,但肺科医生并非如此。患者和护理人员偏好安全性,而肺科医生偏好疗效。临床医生应考虑这些差异,以便在 IPF 治疗决策中更好地让患者参与进来。

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