Centre for Statistics in Medicine, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, University of Oxford, Oxford, UK
Institute of Population Health Sciences, Barts and the London School of Medicine and Dentistry, Queen Mary University of London, London, UK.
BMJ Open. 2021 Jun 28;11(6):e048178. doi: 10.1136/bmjopen-2020-048178.
Prespecified progression criteria can inform the decision to progress from an external randomised pilot trial to a definitive randomised controlled trial. We assessed the characteristics of progression criteria reported in external randomised pilot trial protocols and results publications, including whether progression criteria were specified a priori and mentioned in prepublication peer reviewer reports.
Methodological review.
We searched four journals through PubMed: , , and . Eligible publications reported external randomised pilot trial protocols or results, were published between January 2018 and December 2019 and reported progression criteria. We double data extracted 25% of the included publications. Here we report the progression criteria characteristics.
We included 160 publications (123 protocols and 37 completed trials). Recruitment and retention were the most frequent indicators contributing to progression criteria. Progression criteria were mostly reported as distinct thresholds (eg, achieving a specific target; 133/160, 83%). Less than a third of the planned and completed pilot trials that included qualitative research reported how these findings would contribute towards progression criteria (34/108, 31%). The publications seldom stated who established the progression criteria (12/160, 7.5%) or provided rationale or justification for progression criteria (44/160, 28%). Most completed pilot trials reported the intention to proceed to a definitive trial (30/37, 81%), but less than half strictly met all of their progression criteria (17/37, 46%). Prepublication peer reviewer reports were available for 153/160 publications (96%). Peer reviewer reports for 86/153 (56%) publications mentioned progression criteria, with peer reviewers of 35 publications commenting that progression criteria appeared not to be specified.
Many external randomised pilot trial publications did not adequately report or propose prespecified progression criteria to inform whether to proceed to a future definitive randomised controlled trial.
预先设定的进展标准可以为从外部随机试点试验向确定性随机对照试验推进的决策提供信息。我们评估了外部随机试点试验方案和结果出版物中报告的进展标准的特征，包括进展标准是否事先规定以及在预发表的同行评审报告中是否提及。
方法学综述。
我们通过 PubMed 在四个期刊中进行了搜索： 、 、 、 。符合条件的出版物报告了外部随机试点试验方案或结果，发表于 2018 年 1 月至 2019 年 12 月之间，并报告了进展标准。我们对 25%的纳入出版物进行了数据提取。这里我们报告进展标准的特征。
我们纳入了 160 篇出版物（123 篇方案和 37 项已完成的试验）。招募和保留是促成进展标准的最常见指标。进展标准大多作为明确的阈值报告（例如，达到特定目标；133/160，83%）。不到三分之一纳入了定性研究的计划和已完成的试点试验报告了这些发现将如何有助于进展标准（34/108，31%）。出版物很少说明谁制定了进展标准（12/160，7.5%）或为进展标准提供了理由或依据（44/160，28%）。大多数已完成的试点试验报告了进行确定性试验的意图（30/37，81%），但不到一半的试验严格符合他们所有的进展标准（17/37，46%）。160 篇出版物中有 153 篇（96%）可获得预发表的同行评审报告。153 篇出版物中有 86 篇（56%）的同行评审报告提到了进展标准，35 篇出版物的同行评审员评论说，进展标准似乎没有明确规定。
许多外部随机试点试验出版物没有充分报告或提出预先规定的进展标准，以告知是否推进到未来的确定性随机对照试验。
