PDF(Pubmed)
Abstract：
In \"Explanatory and Pragmatic Attitudes in Therapeutic Trials\", Schwatrz and Lelouch describe two approaches to the design of trials, \"… the first \"explanatory\", the second \"pragmatic\". They explained \"… the biologist may be interested to know whether the drugs differ in their effects … the explanatory approach\". Biologically endpoints might determine whether it was better to give androgen deprivation therapy (ADT) before or after external beam radiation (EBRT) (i.e., does the sequence of treatments matter). Alternatively, if the arms focus on a clinical endpoint, this is considered … \"the pragmatic approach\". An example of a clinically relevant endpoint is overall survival (OS). A real-world example of this are the two randomized controlled trials (RCTs) evaluating the role of prophylactic whole pelvic radiotherapy (WPRT) conducted by the Radiation Therapy Oncology Group (RTOG). RTOG 9413 evaluated possible interactions between the sequence of drugs and volume irradiated, while RTOG/NRG 0924 focuses on OS. There appears to be a common pattern of \"what not to do\", or \"design errors\" made by a number of investigators, that I call the \"three sins\". I posit that the prospects for a well-designed pragmatic RCT are likely to be high if these \"three sins\" are avoided/minimized. The \"three sins\" alluded to are: 1. You can\'t prove something doesn\'t work by treating people who don\'t need the treatment. 2. You can\'t prove something does not work if the treatment is not done properly. 3. You can\'t prove something does not work with an underpowered study.
摘要：
在“治疗试验中的解释和语用态度”中，Schwatrz和Lelouch描述了两种设计试验的方法，\"...第一个\"解释性\",第二个“务实”。他们解释说：“生物学家可能有兴趣知道这些药物的作用是否不同……解释方法。”生物学终点可能决定在外部束辐射（EBRT）之前或之后给予雄激素剥夺治疗（ADT）是否更好（即，治疗的顺序是否重要)。或者，如果手臂专注于临床终点，这被认为是...“务实的方法”。临床相关终点的例子是总生存期(OS)。这方面的一个现实世界的例子是两个随机对照试验（RCT）评估预防性全盆腔放疗（WPRT）的作用由放射治疗肿瘤组（RTOG）进行。RTOG9413评估了药物序列与辐照量之间可能的相互作用，而RTOG/NRG0924专注于操作系统。似乎有一种普遍的“不做什么”的模式，或一些调查人员犯下的“设计错误”，我称之为“三罪”。我认为，如果避免/最小化这些“三罪”，精心设计的实用RCT的前景可能会很高。“三罪”指的是：1。你不能通过治疗不需要治疗的人来证明某些东西不起作用。2.如果治疗不当，你不能证明某些东西不起作用。3.你不能证明一些东西不工作与一个不足的研究。
