A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint.
A randomised controlled trial is not a pilot trial simply because it uses a surrogate endpoint.
复制标题
DOI：
10.1186/s40814-018-0324-2
复制
发表时间：
2018-01-01
影响因子：
1.7
通讯作者：
Eldridge, S M
中科院分区：
文献类型：
--
作者：
Campbell, M J;Lancaster, G A;Eldridge, S M
背景技术背景：有人认为，临床试验的真正终点（或“硬”终点）对临床医生、研究人员和患者都有意义，但仅限于测量健康状况、生存率和成本的终点。其他终点被称为“替代”终点，旨在替代和预测真实终点。许多描述使用替代终点的试验在论文标题中使用了术语“先导”，但其原因是在试验中使用了这些替代终点。此类试验的实施和报告可遵循原始CONSORT声明定义的传统随机对照试验（RCT）的传统模式，采用基于把握度的样本量计算和结果的显著性检验。然而，这违反了CONSORT扩展的试点试验报告指南。主体：我们回顾了替代终点的定义以及替代终点在临床试验中的使用。我们认为在何种程度上可以考虑一个试验，如果它使用一个替代终点和讨论两个例子，说明当前practice.CONCLUSION：使用替代终点的试验只应被描述为“试点”时，一个明确的试验是一个独特的可能性和作者考虑的条件，这将表明是否明确的主要试验是值得和可行的。仅仅因为一项试验使用了替代终点并不能证明它是一项试点试验。
BACKGROUND: It has been argued that true endpoints (or 'hard' endpoints) for clinical trials, which are meaningful to clinicians, researchers and patients alike, are limited to those that measure health status, survival and cost. Other endpoints are termed 'surrogate' endpoints and are intended to substitute and predict the true endpoint.A number of trials that describe using surrogate endpoints use the term 'pilot' in the title of the paper but the reason for this, as related by the authors, is the use of these surrogate endpoints in the trial. The conduct and reporting of such a trial may follow the traditional pattern for a conventional randomised controlled trial (RCT) as defined by the original CONSORT statement, with power-based sample size calculations, and significance tests of the results. However, this is contrary to the guidelines of the CONSORT extension for the reporting of pilot trials.MAIN BODY: We review the definition of a surrogate endpoint and the use of surrogate endpoints in clinical trials. We consider to what extent a trial could be considered a pilot trial if it uses a surrogate endpoint and discuss two examples that illustrate current practice.CONCLUSION: Trials which use surrogate endpoints should only be described as 'pilot' when a definitive trial is a distinct possibility and the authors consider conditions which would indicate whether the definitive main trial was worthwhile and feasible. Simply because a trial uses a surrogate endpoint is not justification for calling it a pilot trial.