University of Liège, Liège, Belgium.
AbbVie Inc, Chicago, IL, USA.
Adv Ther. 2024 Sep;41(9):3678-3705. doi: 10.1007/s12325-024-02923-9. Epub 2024 Aug 6.
Individuals living with Crohn's disease (CD) experience burdensome symptoms. As such, it is important to measure CD symptom severity in clinical research. The goal of this study was to evaluate the content validity, psychometric performance, and score interpretability of a new patient-reported instrument, the Crohn's Symptom Severity (CSS) questionnaire, among adolescents and adults with moderately to severely active CD.
Cognitive debriefing interviews (N = 30; n = 20 adults, n = 10 adolescents) were conducted to evaluate the content validity of the CSS. Additionally, the CSS scores were evaluated for reliability and validity using data from a phase 3 randomized clinical trial of risankizumab (NCT03105128; N = 850). Meaningful within-patient change (MWPC) thresholds were estimated using anchor-based methods.
All interview participants (n = 30/30, 100.00%) reported the CSS was easy to complete and most participants (n = 28/29, 96.55%) reported that the CSS was relevant to their experience of CD. Among the clinical trial subjects (N = 850) the following was found for the CSS: mostly acceptable item-total correlations (0.26-0.79); weak to moderate inter-item correlations (r = 0.07-0.57), good internal consistency (Cronbach's α = 0.76-0.87); intraclass correlation coefficients ranged from 0.48 to 0.70, not consistently exceeding the acceptable range for test-retest reliability (0.70); acceptable convergent validity and known-groups results; and demonstrated sensitivity to change. Analyses supported an MWPC estimate of 6-11 points.
This study supports use of the CSS for measuring CD symptoms and sleep impact among adolescents and adults aged 16 and older with moderately to severely active CD in clinical research.
NCT03105128 (registration date 4 April 2017).
患有克罗恩病（CD）的个体经历着负担沉重的症状。因此，在临床研究中测量 CD 症状的严重程度很重要。本研究的目的是评估一种新的患者报告工具——克罗恩病症状严重程度（CSS）问卷在中重度活动期 CD 的青少年和成年人中的内容有效性、心理测量性能和评分可解释性。
进行认知访谈（N=30；n=20 名成年人，n=10 名青少年）以评估 CSS 的内容有效性。此外，还使用 risankizumab 的 3 期随机临床试验（NCT03105128；N=850）的数据评估 CSS 评分的可靠性和有效性。使用基于锚定的方法估计有意义的患者内变化（MWPC）阈值。
所有访谈参与者（n=30/30，100.00%）均报告 CSS 易于完成，大多数参与者（n=28/29，96.55%）报告 CSS 与他们的 CD 体验相关。在临床试验受试者（N=850）中，CSS 发现：大多数项目总分相关性可接受（0.26-0.79）；项目间相关性较弱至中度（r=0.07-0.57），内部一致性良好（Cronbach's α=0.76-0.87）；组内相关系数范围为 0.48 至 0.70，并不一致地超过测试-重测可靠性的可接受范围（0.70）；具有可接受的收敛有效性和已知组结果；并显示出对变化的敏感性。分析支持 MWPC 估计值为 6-11 分。
这项研究支持在临床研究中使用 CSS 来测量中重度活动期 CD 的青少年和成年人的 CD 症状和睡眠影响。
NCT03105128（注册日期：2017 年 4 月 4 日）。
