应用运动能力和体力活动作为单个和复合终点的心脏康复干预试验:理由,用例和蓝图方法样本大小计算的蓝图方法
Anna Eleonora Carrozzo1, Veronique Cornelissen2, Arne C Bathke3
1Ludwig Boltzmann Institute for Digital Health and Prevention, Salzburg, Austria.
Archives of physical medicine and rehabilitation
|April 15, 2024
概括
结合运动能力和身体活动的新复合终点可能会提高心脏康复试验的统计效率. 这种复合终点 (CE) 可以减少样本大小的需求,但其应用取决于基线数据和干预目标.
科学领域:
- 心脏病学 心脏病学
- 临床试验 临床试验
- 康复医学 康复医学 康复医学
背景情况:
- 心脏康复 (CR) 的干预旨在改善患者的治疗结果.
- 运动能力和体力活动是心脏病患者减少死亡率的关键指标.
- 在CR试验中的当前主要终点可能无法完全捕捉治疗效果.
研究的目的:
- 为基于运动的CR随机对照试验 (RCT) 开发一个复合的初级终点 (CE).
- 评估这一新型CE的统计效率和样本大小要求.
- 探索CE在并行组RCT中的应用.
主要方法:
- 通过结合运动能力和体育活动指标,构想出一个复合终点.
- 进行了对样本大小要求的统计探索.
- 对CE的样本大小需求与单个终点进行了比较,使用两个CR试验的数据.
主要成果:
- 该CE结合了实现的运动能力和身体活动的二分法衡量标准.
- 根据基线比例和干预目标,CE可以增加或减少所需的样本大小.
- 在特定情景中,观察到样本大小减少高达55%或70%.
结论:
- 复合终点为基于运动的CR试验中优化样本大小提供了一个潜在的策略.
- 在实施CE时,试验人员应仔细考虑基线特征和干预目标.
- 在减少样本大小方面,CE的实用性取决于上下文,需要仔细评估.
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