对安慰剂治疗患者的元分析:MASH随机对照试验中治疗中断率
Matheus Souza1, Marcio J M Amaral1, Luan C V Lima1
1Department of Internal Medicine, Federal University of Rio de Janeiro, Rio de Janeiro, Brazil.
Alimentary pharmacology & therapeutics
|January 14, 2025
概括
随机对照试验 (RCT) 中代谢功能障碍相关的脂肪肝炎 (MASH) 的脱落是显著的,主要是由患者的选择驱动的. 较长的试验持续时间和较晚的阶段与更高的学率有关,这影响了研究功率.
科学领域:
- 临床试验 临床试验
- 肝病学 肝病学是一种肝病学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 放弃是随机对照试验 (RCT) 的常见问题,可能会损害统计能力和随机化平衡.
- 代谢功能障碍相关的脂肪肝炎 (MASH) 是一个日益严重的健康问题,需要有效的治疗方法和严格的临床试验评估.
研究的目的:
- 在MASH RCT中量化脱学率.
- 为了确定影响中断的因素,特别是在安慰剂治疗的参与者中.
主要方法:
- 在PubMed和Cochrane数据库中进行了系统的搜索,以寻找与安慰剂手臂的 2-4 期MASH RCT.
- 退学被定义为患者在治疗完成之前从治疗意图分析中退出.
- 用一个通用的线性混合模型来估计聚合的学率.
主要成果:
- 对60个MASHRCT (3230名安慰剂参与者) 的分析显示,整体学率为11.06%.
- 与第二阶段试验相比,在3-4期试验中,中断率更高,并且随着治疗持续时间的延长而增加.
- 患者选择 (4.06%) 是脱学的主要原因,其次是不良事件 (2.41%) 和失去随访 (1.79%).
结论:
- 在MASH试验中,安慰剂弃率很大,并且受到患者选择的重大影响.
- 试验阶段和治疗持续时间是考虑在未来MASH临床试验中准确样本大小计算的关键因素.
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