当临床试验的特征需要每项方案以及治疗意图的结果来得出可靠的结论时:三个例子
David E Scheim1, Colleen Aldous2, Barbara Osimani3
1US Public Health Service, Commissioned Corps, Inactive Reserve, Blacksburg, VA 24060, USA.
Journal of clinical medicine
|June 10, 2023
概括
每个协议 (PP) 分析可能会揭示真正的医疗干预益处,当协议遵守率低时,与意图治疗 (ITT) 分析不同. 三项随机临床试验 (RCT) 突出了PP和ITT结果之间的差异,强调了数据透明度的需求.
科学领域:
- 临床试验方法论 临床试验方法论
- 医疗干预的有效性 医疗干预的有效性
- 研究中的数据透明度
背景情况:
- 治疗意图 (ITT) 分析是随机临床试验 (RCT) 的标准,但如果合规性很低,可能会掩盖现实世界的益处.
- 每项协议 (PP) 分析只考虑那些遵守试验协议的参与者,这可能会更清楚地了解干预措施的真实效果.
- ITT和PP分析之间的差异可能会出现,特别是在高不合规率或数据报告问题研究中.
研究的目的:
- 举例来说明,每条协议 (PP) 分析比治疗意图 (ITT) 分析更准确地反映了医疗干预的好处.
- 突出协议不遵守对临床试验结果解释的影响.
- 强调数据透明度在解决ITT和PP结果之间的差异方面的重要性.
主要方法:
- 对三项随机临床试验 (RCT) 进行了审查,其协议遵守程度各不相同.
- 对医疗干预的意图治疗 (ITT) 和每协议 (PP) 分析结果的比较.
- 检查临床研究中的数据报告实践和可访问性.
主要成果:
- 第一个RCT:结肠镜查显示ITT分析的边际益处 (42%的遵守),但PP分析的癌症死亡率减少了50%.
- 第二个RCT:一种COVID-19药物通过PP分析显示死亡率降低了十倍,而ITT分析则显示了微不足道的益处.
- 第三次RCT:另一种COVID-19药物通过ITT没有显著的益处;尽管存在不合规问题,但死亡/住院病人的PP数据没有披露.
结论:
- 每个协议 (PP) 分析可以揭示意图治疗 (ITT) 分析所遗漏的显著的医疗干预疗效,特别是在遵守性较低的情况下.
- 报告协议合规性和数据访问的不一致性阻碍了对试验结果的准确解释.
- 确保数据的透明度对于验证发现和理解医疗干预的真正影响至关重要,当PP和ITT的结果分歧时.
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