在干预试验中应应用哪些终点? -:从针对药物的作用机制的单一单维评估到多组件措施和多域复合材料
Felix Eckstein1,2,3, Tanja Stamm2,4, Jamie Collins5
1Research Group for Musculoskeletal Imaging, Center for Anatomy and Cell Biology, Paracelsus Medical University, Salzburg, Austria.
选择正确的骨关节炎评估是研究的关键. 将多种成像和临床结果结合成复合终点提供了灵活性,但需要仔细的统计规划.
科学领域:
- 骨关节炎的研究研究.
- 医学成像医学成像
- 临床试验的设计
背景情况:
- 许多结构/成像和临床终点可用于骨关节炎研究.
- 对于特定研究,这些评估的最佳选择和应用仍未确定.
- 目前的做法往往涉及分析多个评估一起或创建复合终点.
研究的目的:
- 审查与骨关节炎研究中的多组件和复合终点相关的概念和挑战.
- 提供来自当前骨关节炎成像研究的例子.
- 讨论如何将各种评估整合到复杂的结果指标中.
主要方法:
- 使用PubMed和其他数据库进行了叙事文献搜索.
- 与现场专家进行非正式商.
- 识别的概念和发现被组织成一个综合框架.
主要成果:
- 单个成像评估可以是单维或多维的.
- 综合测量可以通过整合跨解剖位置的评估或结合异质评估类型 (多组件/复合终点) 来形成.
- 权重,标准化和临床实用性对于多组件/复合终点至关重要,在骨关节炎成像中提供了例子.
结论:
- 在骨关节炎研究中,多组件/复合终点的可能性是广泛的.
- 战略研究设计和适当的统计方法对于探索和验证这些终点至关重要.
- 普遍的方法不太可能成功;成像评估应根据药物机制和患者特征进行量身定制.
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