背景:在随机临床试验(RCT)中具有代表性的人群可以提高试验结果的外部有效性和普遍性。研究RCT登记人群和真实世界人群在血流感染(BSI)方面的差异的数据有限。
目的:我们进行了范围审查,旨在审查评估BSIRCT人群普遍性的研究,确定系统代表性不足的亚组,并探索提高未来RCT外部有效性的方法。
方法:MEDLINE,Embase,并在Cochrane图书馆数据库中搜索与外部有效性或泛化性相关的术语,BSI,以及截至2023年8月1日发表的论文中的临床试验。比较入选和非入选患者的研究,或包括在BSIRCT背景下讨论外部有效性或普遍性的论文。
背景:最终审查中包含了16篇论文。五名比较了来自同一来源人群的RCT注册和非注册参与者。在所有研究中,两组之间存在显着差异。未纳入的患者的合并症负担更大,包括死亡率在内的结局持续更差。我们确定了提高RCT人群普遍性的几个障碍,并概述了减少这些障碍的潜在方法,如替代/简化的同意程序,简化资格标准和后续程序,基于配额的抽样技术,并确保地点和研究团队选择的多样性。
结论:BSI随机对照试验中的研究队列并不代表一般的BSI患者群体。随着我们越来越多地在传染病领域采用大型务实试验,重要的是要认识到最大限度地推广的重要性,以确保我们的研究结果与我们的患者直接相关.
BACKGROUND: Having a representative population in randomized clinical trials (RCTs) improves external validity and
generalizability of trial results. There are limited data examining differences between RCT-enrolled and real-world populations in bloodstream infections (BSI).
OBJECTIVE: We conducted a scoping
review aiming to
review studies assessing
generalizability of BSI RCT populations, to identify sub-groups that have been systematically under-represented and to explore approaches to improve external validity of future RCTs.
METHODS: MEDLINE, Embase, and Cochrane Library databases were searched for terms related to external validity or generalizability, BSI, and clinical trials in papers published up to 1 August 2023. Studies comparing enrolled versus nonenrolled patients, or papers discussing external validity or
generalizability in the context of BSI RCTs were included.
BACKGROUND: Sixteen papers were included in the final
review. Five compared RCT-enrolled and nonenrolled participants from the same source population. There were significant differences between the two groups in all studies, with nonenrolled patients having a greater comorbidity burden and consistently worse outcomes including mortality. We identified several barriers to improving
generalizability of RCT populations and outlined potential approaches to reduce these barriers, such as alternative/simplified consent processes, streamlining eligibility criteria and follow-up procedures, quota-based sampling techniques, and ensuring diversity in site and study team selection.
CONCLUSIONS: Study cohorts in BSI RCTs are not representative of the general BSI patient population. As we increasingly adopt large pragmatic trials in infectious diseases, it is important to recognize the importance of maximizing generalizability to ensure that our research findings are of direct relevance to our patients.