治疗意图和每项方案分析:差异和相似之处
Javier Molero-Calafell1, Andrea Burón2, Xavier Castells3
1Department of Epidemiology and Evaluation, Hospital del Mar (HMar), Barcelona, Spain; Hospital del Mar Research Institute (IMIM), Barcelona, Spain; Preventive Medicine and Public Health Training Unit HMar-UPF-ASPB (HMar - Pompeu Fabra University - Agència de Salut Pública de Barcelona), Barcelona, Spain.
了解随机试验中的意图治疗 (ITT) 和每协议 (PP) 分析至关重要. ITT将所有随机选择的患者进行比较,而PP仅分析那些遵守协议的人,每个人都有不同的优势和局限性.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 流行病学 流行病学
背景情况:
- 随机试验采用解释性或务实的方法.
- 解释性试验在最佳条件下评估疗效,通常使用每协议 (PP) 分析.
- 实用试验在现实环境中评估有效性,优先考虑治疗意图 (ITT) 分析.
研究的目的:
- 阐明随机试验中意图治疗 (ITT) 和每协议 (PP) 分析的不同目标,优势和局限性.
- 要突出遵守如何影响ITT和PP分析的有效性和解释.
- 引入先进的统计方法来解决试验分析中的偏见.
主要方法:
- 讨论ITT和PP分析的概念基础.
- 检查遵守和不遵守如何影响试验结果和解释.
- 引入一般化方法 (g-方法),如反向概率权重.
主要成果:
- 由于不遵守,PP分析有可能失去随机化的好处,并引入随机化后的混.
- ITT分析可能会稀释治疗效果,并面临不同坚持的适用性问题.
- 无论是ITT还是PP分析,都容易因缺少数据而产生选择偏差.
结论:
- ITT和PP分析是互补的,但具有固有的局限性,受遵守的影响.
- 随机化后的混和选择偏差可能会影响试验有效性.
- 像g方法这样的先进统计方法可以减轻ITT和PP分析中的偏差.
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