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患者报告结果测量的谬论:为什么他们经常无法检测出临床重要差异
Nathan N O'Hara1, Dane J Brodke2, Ashley E Levack3
1Center for Orthopaedic Injury Research and Innovation, Department of Orthopaedics, University of Maryland School of Medicine, Baltimore, Maryland.
JB & JS open access
|September 9, 2025
概括
像非联合手术这样的二元结果更有可能在骨科临床试验中检测出显著差异,而不是连续的患者报告结果. 这突显了在某些研究中使用患者报告的结果来检测有意义的治疗效应的关键局限性.
科学领域:
- 整形外科手术 整形外科手术
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
背景情况:
- 患者报告的结果测量 (PROM) 在骨科研究中越来越多地使用.
- 然而,PROM通常无法检测临床试验中的临床上有意义的差异.
- 这项研究比较了二进制与连续PROM检测显著差异的能力.
研究的目的:
- 评估骨科研究是否更有可能通过二元结果 (例如,非联合手术) 或连续患者报告的结果来检测临床重要差异.
- 为了比较二进制与连续结果在检测治疗效应的统计能力.
主要方法:
- 模拟了一个假设的临床试验,比较两种治疗骨骨折的治疗方法.
- 假设治疗组的非工会率为10%,对照组为20%.
- 模拟了400名患者的1万项试验,分析了非联合手术率和患者报告结果测量信息系统 (PROMIS) 的身体功能得分.
主要成果:
- 非联合手术的二元结果在80.9%的模拟试验中具有统计学意义.
- 持续的PROMIS身体功能得分仅在4.2%的试验中具有统计学意义.
- 观察到检测结果的显著差异 (p < 0.001).
结论:
- 使用PROM的研究可能无法检测到统计学上显著的差异,当临床上重要的结果发生在少数患者中时.
- 整形外科医生和研究人员在设计和评估研究时应考虑这一局限性.
- 意识到PROM的局限性对于准确的治疗比较至关重要.
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