通过例行收集的数据进行终点评估,产生与随机对照试验数据可比的估计:对预防摔倒伤害的集群随机试验的分析
David A Ganz1, Erich J Greene2, Nancy K Latham3
1Department of Medicine, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA; Geriatric Research, Education and Clinical Center, Veterans Affairs Greater Los Angeles Healthcare System, Los Angeles, CA, USA; Health Care Division, RAND, Santa Monica, CA, USA.
Journal of clinical epidemiology
|February 12, 2025
概括
在试验中,常规收集的数据 (RCD) 显示了类似的治疗效果估计,但可信度间隔更宽. 验证RCD结果需要测量临床试验终点的准确性不确定性.
科学领域:
- 临床试验方法论 临床试验方法论
- 医疗保健服务研究 医疗服务研究
- 在医疗保健中的数据科学.
背景情况:
- 常规收集的数据 (RCD) 越来越多地用于随机试验.
- 评估RCD有效性和精度的方法是有限的.
- 开发了一种方法来测量RCD有效性,相对精度和不确定性.
研究的目的:
- 开发和应用一种方法来测量RCD衍生结果的有效性和相对精度.
- 量化临床试验中与RCD相关的不确定性.
- 在STRIDE试验中,将RCD结果与常规确定的结果进行比较.
主要方法:
- 重新分析了"减少伤害和培养老年人信心的策略" (STRIDE) 试验的数据.
- 92%的参与者与RCD联系在一起.
- 估计的危险比率 (HRs) 和使用原始数据和RCD的置信区间 (CI).
- 计算HRs的有效率比率和相对精度的信任限值比率 (CLRs) 的比率.
- 使用引导来估计不确定性.
主要成果:
- 在RCD中,干预的HR为0.91 (95%CI:0.78-1.07),而在原始数据中为0.92 (95%CI:0.80-1.06).
- HRs的比率为1.00 (95% CI:0.89-1.11),表明类似的有效性.
- 临床相关反应的比为1.03 (95% CI:0.96-1.06),表明精度略低.
- 对于HRs的置信区间是RCD的约3倍.
- 随着随访时间的延长,相对精度有所提高.
结论:
- 仅依赖RCD就产生了STRIDE中治疗效果的类似点估计和CI.
- 在RCD衍生结果的有效性估计方面存在重大不确定性.
- 未来的努力应优先考虑对临床试验终点RCD有效性的测量不确定性.
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