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采用离散选择实验技术的炎症性肠病患者治疗偏好:InPuT 研究。

Inflammatory Bowel Disease Patients' Treatment Preferences Using a Discrete Choice Experiment Technique: The InPuT Study.

机构信息

Gastroenterology Practice am Bayerischen Platz, Innsbrucker Str. 58, 10825, Berlin, Germany.

Ingress-Health HWM GmbH, Alter Holzhafen 19, 23966, Wismar, Germany.

出版信息

Adv Ther. 2022 Jun;39(6):2889-2905. doi: 10.1007/s12325-022-02143-z. Epub 2022 Apr 22.

Abstract

INTRODUCTION

The aim of this study was to investigate patients' preferences regarding the evolving treatment landscape in Crohn's disease (CD) and ulcerative colitis (UC) based on a discrete choice experiment.

METHODS

Eligible patients (aged 18 years or older) had a confirmed diagnosis of CD or UC and were willing and able to participate in telephone interviews. The survey design is based on a prior literature review, a pilot study, and clinical expert discussions. Preferences related to clinical and practical features of advanced therapies, like tumor necrosis factor alpha inhibitors, anti-integrins, anti-interleukins, and Janus kinase inhibitors, were assessed. Patients were asked to choose between two different hypothetical treatment alternatives visualized in up to 11 choice scenarios. Based on these choices, the relative importance of treatment characteristics was derived from regression coefficients estimated by a conditional logit model.

RESULTS

Of the 291 patients included, 219 (75%) were eligible for this analysis. Among the evaluated attributes in CD, 1-year remission rate was ranked highest, with 42.3% relevance for the overall decision. The second most important attribute was the frequency of serious adverse events (AE) (25.1%), followed by sustained remission over 2 years (17.8%). Lower importance was assigned to the administration mode (14.6%) and none to the frequency of non-serious AE (0.1%). In UC, preferences were driven by efficacy (25.3% for mucosal healing; 23.4% for corticosteroid-free remission) and the frequency of serious AE (18.3%), followed by the administration mode (18.1%). Also, non-serious AE were classified as relevant factors for decision-making (10.7%), while maintaining remission for at least 2 years showed no significant impact (4.4%).

CONCLUSION

For both indications, efficacy outcomes were rated most important, followed by the frequency of serious AE. Variations were mainly found in the evaluation of non-serious AE and sustained remission. Considering patient preferences may improve the effectiveness of available therapies for moderate to severe CD and UC.

摘要

介绍

本研究旨在通过离散选择实验调查克罗恩病(CD)和溃疡性结肠炎(UC)患者对不断发展的治疗前景的偏好。

方法

符合条件的患者(年龄在 18 岁或以上)确诊为 CD 或 UC,愿意并能够接受电话访谈。调查设计基于先前的文献综述、一项试点研究和临床专家讨论。评估了抗肿瘤坏死因子-α抑制剂、抗整合素、抗白细胞介素和 Janus 激酶抑制剂等先进疗法的临床和实际特征相关的偏好。患者被要求在最多 11 个选择场景中选择两种不同的假设治疗方案。基于这些选择,通过条件逻辑回归模型估计的回归系数得出治疗特征的相对重要性。

结果

在 291 名纳入的患者中,有 219 名(75%)符合本分析条件。在 CD 评估的属性中,1 年缓解率排名最高,对整体决策的相关性为 42.3%。第二个最重要的属性是严重不良事件(AE)的频率(25.1%),其次是持续缓解超过 2 年(17.8%)。给药方式的重要性较低(14.6%),非严重 AE 的频率(0.1%)则无重要性。在 UC 中,偏好受疗效驱动(黏膜愈合 25.3%;无皮质类固醇缓解 23.4%)和严重 AE 的频率(18.3%),其次是给药方式(18.1%)。此外,非严重 AE 被归类为决策的相关因素(10.7%),而至少维持 2 年缓解则没有显著影响(4.4%)。

结论

对于这两种疾病,疗效结果被评为最重要的,其次是严重 AE 的频率。在非严重 AE 和持续缓解的评估中主要存在差异。考虑患者的偏好可能会提高现有治疗中度至重度 CD 和 UC 的有效性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/74ee/9122873/9903825893a4/12325_2022_2143_Fig1a_HTML.jpg

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