性结肠炎患者的经验:概念模型和患者报告的结果措施的综述
Chong Kim1, Fiona L Brown2, Caroline Burk3
1HEOR, Gilead Sciences, Foster City, CA, USA. Chong.Kim9@gilead.com.
对性结肠炎 (UC) 患者报告的结果措施捕捉了关键症状和影响,但需要进一步验证. 这项研究评估了现有的措施,以确保它们适合临床研究的目的.
科学领域:
- 胃肠病学 胃肠病学
- 临床研究 临床研究
- 患者报告的结果
背景情况:
- 性结肠炎 (UC) 显著影响日常功能和与健康相关的生活质量 (HRQoL).
- 准确评估UC症状和影响对于有效的临床研究至关重要.
- 患者报告结果 (PRO) 措施是捕捉UC患者体验的重要工具.
研究的目的:
- 识别和描述症状及其对成人UC患者日常功能和HRQoL的影响.
- 评估现有的PRO措施是否适用于UC临床研究.
- 评估已识别的PRO措施的概念覆盖范围和心理特征.
主要方法:
- 从文献评论中开发了UC症状和影响的概念模型.
- 从文献,临床试验数据库和监管机构提交的文件中确定了PRO措施.
- 根据疾病特异性和使用情况进行概念分析的PRO措施,根据概念模型评估内容有效性.
- 使用FDA指南和COSMIN标准评估心理测量特性.
主要成果:
- 开发了一个概念模型,包括52个症状概念和92个多维影响.
- 在65个已识别的PRO措施中,有8个经过了详细的概念分析.
- 一些PRO措施,包括炎症性肠病问卷和UC-PRO模块,显示出良好的内容有效性.
- 该UC-PRO标志和症状测量完全符合FDA和COSMIN的内容有效性和大多数心理测量属性的标准.
结论:
- 现有的PRO措施捕捉了UC患者的相关概念.
- 所有审查的PRO措施都需要进一步的心理测量评估,以确认它们是否适合目的.
- 这项研究强调了在UC研究中需要强有力的和经过验证的PRO措施.
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