为什么大多数受访者分析都是误导性的
Aleksandra Turkiewicz1, Marius Henriksen2, Jos Runhaar3
1Clinical Epidemiology Unit, Orthopedics, Clinical Sciences Lund, Lund University, Lund, Sweden.
Osteoarthritis and cartilage
|December 23, 2025
概括
在骨关节炎试验中的响应者分析是误导性的. 模拟显示这些方法低估了治疗的有效性,并错误地表明了变化,即使是高效的治疗.
科学领域:
- 临床试验方法论 临床试验方法论
- 骨关节炎研究 骨关节炎研究
- 疼痛管理统计 疼痛管理统计
背景情况:
- 响应者分析在骨关节炎随机对照试验 (RCT) 中很常见.
- 这些分析通常依赖于从基线观察到的自我报告疼痛的变化.
- 方法论文献表明,这些应答者分析可能具有误导性.
研究的目的:
- 为了说明误导性响应者的问题的程度,使用模拟进行了响应分析.
- 评估自然疼痛波动对响应者分析结果的影响.
主要方法:
- 使用现实的假设生成个人疼痛轨迹 (正常分布,平均VAS 45,SDs).
- 来自RCT的模拟数据具有不同的真实治疗效果 (0-15VAS点) 和响应者比例 (0%或100%).
- 应用于模拟试验数据的标准响应者分析方法.
主要成果:
- 从基线观察到的疼痛变化并不等同于由于自然波动而导致的治疗反应.
- 即使有15点的VAS治疗效果和0%的安慰剂反应,分析表明80%的响应者与安慰剂中的50%相比.
- 这低估了治疗疗效,错误地暗示了治疗效果的异质性.
结论:
- 基于视觉模拟尺度 (VAS) 疼痛与基线的变化进行响应者分析,应停止对平行组RCT进行治疗.
- 对于其他波动的患者报告结果 (症状,功能,总体评估) 使用基线变化的响应者标准,由于类似的局限性,需要仔细审查.
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