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关于“具有定量反应的剂量递增试验设计的评论”。

Commentary on 'Designs for dose-escalation trials with quantitative responses'.

机构信息

INSERM, Université Pierre et Marie Curie, Paris VI, Place Jussieu, 75005 Paris, France.

出版信息

Stat Med. 2009 Dec 30;28(30):3745-50; discussion 3759-60. doi: 10.1002/sim.3742.

Abstract

Bailey (Statist. Medi 2009; this issue) puts the focus on efficiency in dose-finding studies and shows that some quite simple modifications to much current experimentation can lead to significant efficiency gains. Efficiency has not been the main focus of interest in this field, in particular when dealing with situations requiring other than healthy volunteers. Dr Bailey's results are important, both in theory and in practice and also raise the question as to what ought to be the guiding principle to statistical workers in this field. Safety, although escaping any simple definition, has mostly guided experimentation in dose-finding studies; one reason being that the concept is one on which clinicians and statisticians can come to some broad agreement. Finding such agreement may still be the single major difficulty in these studies, alongside the establishment of criteria that all of the scientists involved believe are the most appropriate and useful ones.

摘要

贝利(Statist. Med. 2009;本期)关注剂量探索研究中的效率,并表明对当前许多实验进行一些相当简单的修改就能显著提高效率。在这个领域,效率并不是主要关注的焦点,尤其是在处理需要非健康志愿者的情况时。贝利博士的结果无论在理论上还是实践中都很重要,也提出了一个问题,即统计工作者在这个领域应该遵循什么指导原则。安全性虽然没有一个简单的定义,但在剂量探索研究中大多指导着实验;原因之一是这个概念是临床医生和统计学家可以达成一些广泛共识的概念。在这些研究中,找到这样的共识可能仍然是最大的困难之一,此外还需要建立所有参与科学家都认为最合适和最有用的标准。

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