Welsing Paco M, Oude Rengerink Katrien, Collier Sue, Eckert Laurent, van Smeden Maarten, Ciaglia Antonio, Nachbaur Gaelle, Trelle Sven, Taylor Aliki J, Egger Matthias, Goetz Iris
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, The Netherlands.
Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, The Netherlands.
J Clin Epidemiol. 2017 Oct;90:99-107. doi: 10.1016/j.jclinepi.2016.12.022. Epub 2017 May 11.
Results from pragmatic trials should reflect the comparative treatment effects encountered in patients in real-life clinical practice to guide treatment decisions. Therefore, pragmatic trials should focus on outcomes that are relevant to patients, clinical practice, and treatment choices. This sixth article in the series (see Box) discusses different types of outcomes and their suitability for pragmatic trials, design choices for measuring these outcomes, and their implications and challenges. Measuring outcomes in pragmatic trials should not interfere with real-world clinical practice to ensure generalizability of trial results, and routinely collected outcomes should be prioritized. Typical outcomes include mortality, morbidity, functional status, well-being, and resource use. Surrogate endpoints are typically avoided as primary outcome. It is important to measure outcomes over a relevant time horizon and obtain valid and precise results. As pragmatic trials are often open label, a less subjective outcome can reduce bias. Methods that decrease bias or enhance precision of the results, such as standardization and blinding of outcome assessment, should be considered when a high risk of bias or high variability is expected. The selection of outcomes in pragmatic trials should be relevant for decision making and feasible in terms of executing the trial in the context of interest. Therefore, this should be discussed with all stakeholders as early as feasible to ensure the relevance of study results for decision making in clinical practice and the ability to perform the study.
实用性试验的结果应反映现实临床实践中患者所遇到的比较治疗效果，以指导治疗决策。因此，实用性试验应关注与患者、临床实践及治疗选择相关的结局。本系列的第六篇文章（见方框）讨论了不同类型的结局及其对实用性试验的适用性、测量这些结局的设计选择，以及它们的影响和挑战。在实用性试验中测量结局不应干扰现实世界的临床实践，以确保试验结果的可推广性，并且应优先考虑常规收集的结局。典型的结局包括死亡率、发病率、功能状态、幸福感和资源利用情况。通常避免将替代终点作为主要结局。在相关的时间范围内测量结局并获得有效且精确的结果很重要。由于实用性试验通常是开放标签的，一个主观性较低的结局可以减少偏倚。当预期存在高偏倚风险或高变异性时，应考虑采用减少偏倚或提高结果精度的方法，如结局评估的标准化和盲法。实用性试验中结局的选择应与决策相关，并且在感兴趣的背景下进行试验时应切实可行。因此，应尽早与所有利益相关者进行讨论，以确保研究结果与临床实践决策的相关性以及开展研究的能力。
