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[ADA2012]患者依从性在临床试验中的重要意义
——爱因斯坦大学医学院内分泌科Jeffrey S. Gonzalez教授
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作者:J.S.Gonzalez 2012/6/13 16:28:00    加入收藏
内容概要:Gonzalez教授:在整个试验中仔细收集依从性的数据,并在整个调查过程中对依从性进行监测,有助于我理解你比较一种干预与另一种干预之效果的意向性分析的结果,这对公众健康具有非常重要的意义。

  <International Diabetes>: How can collecting adherence data throughout a study bridge the gap between intention-to-treat and per-protocol? Can you explain that for us?
  Dr Gonzalez: Collecting careful adherence data throughout the trial and monitoring adherence over the course of the investigation in both conditions can allow me to then look at your intention-to-treat analysis where you compare the effect of being assigned to receive one or the other intervention (and that has public health implications), but then I think the benefit compared to a per-protocol analysis is that you don’t necessarily have to throw out any of your data. You can look at whether ones level of adherence impacts the effects that are obtained by the drug under investigation or maybe it’s adherence to a particular behavior protocol or whatever it. If you can quantify someone’s level of adherence and use that as a variable that may moderate the treatment’s effectiveness, I think that can add a lot of information about what effect can be expected out in the real world where adherence is going to be a bigger problem than it was in your trial where you have tightly controlled for factors that might limit a patient’s ability to adhere.

  《国际糖尿病》:您能否给我们解释一下,我们应该如何在整个研究中收集有关依从性的数据,以缩小意向性分析和符合方案分析之间的差距?
  Gonzalez教授:在整个试验中仔细收集依从性的数据,并在整个调查过程中对依从性进行监测,有助于我理解你比较一种干预与另一种干预之效果的意向性分析的结果,这对公众健康具有非常重要的意义。我认为与符合方案分析相比,意向性治疗的优势在于不需要对数据进行处理,也就是剔除某些数据。你可以确定药物治疗时依从性的高低、对某种特殊行为的依从性或其他是否会影响所观察的药物的疗效。如果你可以对依从性水平进行量化,并将其作为有可能会降低治疗疗效的变量,这将为我们提供哪些效应可以被预期排除的大量信息,因为在试验中你能够严格控制影响依从性的因素,与之相比,现实世界中依从性则是个很大的问题。

  <International Diabetes>: Whenever you are looking at statistics for people who had adhered though, they are always going to be tainted numbers because you are always going to be looking at the “healthy adherers”.
  Dr Gonzalez: That’s right. I think that is part of the problem with a per-protocol analysis. The way a lot of people handle a per-protocol analysis is that they throw the non-adherent people out of their experimental trial and only focus on the adherers. But in the control condition, they compare them to everyone so this analysis I am presenting is an alternative approach where you don’t throw anyone out but you ask the question of whether one’s adherence influenced the effect that was obtained.
  In the DPP project I mentioned in my talk, within the metformin arm they looked at whether adherence was associated with benefit in terms of decreased risk of developing diabetes and then they were able to show that that effect was not found in the placebo group. You have to be worried about or cautious about this “healthy adherer” effect, but that was a nice demonstration that said adherence is the same thing in both of these conditions and people are randomly assigned and we are keeping all of them in there so we can be confident about balancing but now you see it, and now you don’t. In metformin there is an adherence effect and in placebo there is not. It depends what you are adhering to. The more you adhere, the better benefit but it is dependent on what you are taking.

  《国际糖尿病》:在对依从性较高的人群进行统计时,这些数据通常并不真实,因为你通常会把一部分健康的依从者纳入其中。
  Gonzalez教授:是的。我认为这是符合方案分析的问题之所在。很多人进行符合方案分析时会将依从性较差者从其试验中剔除,只关注或分析那些依从性较高的受试者。但是在对照条件下,他们会将其与其他所有人进行比较,因此我所提到的这种分析是一种很好的替代方法。采用该方法你无需剔除任何参与的受试者,只需要询问他们问题,了解依从性是否会影响获得的疗效。
  我演讲中所提到的DPP计划,在二甲双胍治疗组他们观察了依从性是否与糖尿病发病风险降低的获益有相关性,然后他们发现该效应并未出现于安慰剂组。你必须对“健康依从”效应持谨慎态度,这是对于那些认为这些随机分组的人群依从性是相同的人是一个很好的范例。我们对两组间的平衡有充分的信心。但是现在你看到了,二甲双胍组存在依从性效应而安慰剂组则没有。这主要取决于你所依从的是什么。你的依从性越高,获益将越多,但是这取决于你所采取的措施。

  <International Diabetes>: Do we see this kind of result a lot?
  Dr Gonzalez: Not a lot. Part of the reason for this whole symposium was to encourage people engaged in clinical trials and research who may not be focusing on behavioral factors, adherence or treatment satisfaction. If you take these factors into account, even if your objective is just does my drug beat that competitor’s drug or are they equivalent, for instance, taking account of this information will give you a better evaluation of that question than the two choices of intention-to-treat or per-protocol. If you have detailed information on adherence, you can use it in the evaluation of your effects.

  《国际糖尿病》:我们是否看到了很多这样的结果?
  Gonzalez教授:并不是很多。进行这样一场研讨会的一部分目的是鼓励那些可能并不关注行为因素、依从性或治疗满意度的人们从事临床试验和研究。即使你的目的仅仅是为了确定自己的药物是否优于竞争者的药物,或是两者相当,如果考虑这些因素的话,与意向性治疗或符合方案相比,你将能更好地评估这个问题。如果你能够获得依从性的详细信息,便能够利用它来评估试验的效果。
 



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