Staibano Phillip, McKechnie Tyler, Thabane Alex, Olteanu Daniel, Nanji Keean, Zhang Han, Lunny Carole, Au Michael, Gupta Michael K, Pasternak Jesse D, Parpia Sameer, Young Jem Ted, Bhandari Mohit
Division of Otolaryngology-Head and Neck Surgery, Department of Surgery, McMaster University, Hamilton, Ontario, Canada.
Department of Health Research Methodology, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada.
PLoS One. 2024 Dec 12;19(12):e0303315. doi: 10.1371/journal.pone.0303315. eCollection 2024.
Randomized-clinical trials (RCTs) are the gold-standard for comparing health care interventions, but can be limited by early termination, feasibility issues, and prolonged time to trial reporting. Adaptive clinical trials (ACTs), which are defined by pre-planned modifications and analyses that occur after starting patient recruitment, are gaining popularity as they can streamline trial design and time to reporting. As adaptive methodologies continue to be adopted by researchers, it will be critical to develop a risk of bias tool that evaluates the unique methodological features of ACTs so that their quality can be improved and standardized for the future. In our proposed methodological review, we will develop a list of risk of bias items and concepts, so that a risk of bias tool specific to ACTs can be developed.
We will perform a systematic database search to capture studies that have proposed or reviewed items pertaining to methodological risk, bias, and/or quality in ACTs. We will perform a comprehensive search of citation databases, such as Ovid MEDLINE, EMBASE, CENTRAL, the Cochrane library, and Web of Science, in addition to multiple grey literature sources to capture published and unpublished literature related to studies evaluating the methodological quality of ACTs. We will also search methodological registries for any risk of bias tools for ACTs. All screening and review stages will be performed in duplicate with a third senior author serving as arbitrator for any discrepancies. For all studies of methodological quality and risk of bias, we will extract all pertinent bias items, concepts, and/or tools. We will combine conceptually similar items in a descriptive manner and classify them as referring to bias or to other aspects of methodological quality, such as reporting. We will plan to generate pertinent risk of bias items to generate a candidate tool that will undergo further refinement, testing, and validation in future development stages.
This review does not require ethics approval as human subjects are not involved. As mentioned previously, this study is the first step in developing a tool to evaluate the risk of bias and methodological quality of ACTs. The findings of this review will inform a Delphi study and the development of a risk of bias tool for ACTs. We plan on publishing this review in a peer-reviewed journal and to present these findings at international scientific conferences.
随机临床试验（RCTs）是比较医疗保健干预措施的金标准，但可能受到早期终止、可行性问题以及试验报告时间延长的限制。适应性临床试验（ACTs）是指在开始招募患者后进行预先计划的修改和分析，因其可以简化试验设计和报告时间而越来越受欢迎。随着研究人员继续采用适应性方法，开发一种评估ACTs独特方法学特征的偏倚风险工具至关重要，以便未来能够提高其质量并使其标准化。在我们提议的方法学综述中，我们将制定一份偏倚风险项目和概念清单，以便开发一种特定于ACTs的偏倚风险工具。
我们将进行系统的数据库搜索，以获取已提出或审查与ACTs方法学风险、偏倚和/或质量相关项目的研究。除了多个灰色文献来源外，我们还将对文献数据库进行全面搜索，如Ovid MEDLINE、EMBASE、CENTRAL、Cochrane图书馆和Web of Science，以获取与评估ACTs方法学质量的研究相关的已发表和未发表文献。我们还将搜索方法学注册库，查找ACTs的任何偏倚风险工具。所有筛选和审查阶段将由两人重复进行，如有任何差异，由第三位资深作者担任仲裁。对于所有方法学质量和偏倚风险研究，我们将提取所有相关的偏倚项目、概念和/或工具。我们将以描述性方式合并概念相似的项目，并将它们分类为涉及偏倚或方法学质量的其他方面，如报告。我们计划生成相关的偏倚风险项目，以生成一个候选工具，该工具将在未来的开发阶段进行进一步完善、测试和验证。
本综述不需要伦理批准，因为不涉及人类受试者。如前所述，本研究是开发评估ACTs偏倚风险和方法学质量工具的第一步。本综述的结果将为德尔菲研究和ACTs偏倚风险工具的开发提供信息。我们计划在同行评审期刊上发表本综述，并在国际科学会议上展示这些结果。
