QOL Office, Level 6 North, Lifehouse (C39Z), University of Sydney, Sydney, NSW, Australia.
Mayo Clinic, Scottsdale, AZ.
Med Care. 2019 May;57 Suppl 5 Suppl 1(Suppl 5 1):S38-S45. doi: 10.1097/MLR.0000000000001111.
Patient-reported outcome (PRO) data may be used at 2 levels: to evaluate impacts of disease and treatment aggregated across individuals (group-level) and to screen/monitor individual patients to inform their management (individual-level). For PRO data to be useful at either level, we need to understand their clinical relevance.
To provide clarity on whether and how methods historically developed to interpret group-based PRO research results might be applied in clinical settings to enable PRO data from individual patients to inform their clinical management and decision-making.
We first differentiate PRO-based decision-making required at group versus individual levels. We then summarize established group-based approaches to interpretation (anchor-based and distribution based), and more recent methods that draw on item calibrations and qualitative research methods. We then assess the applicability of these methods to individual patient data and individual-level decision-making.
Group-based methods provide a range of thresholds that are useful in clinical care: some provide screening thresholds for patients who need additional clinical assessment and/or intervention, some provide thresholds for classifying an individual's level of severity of symptoms or problems with function, and others provide thresholds for meaningful change when monitoring symptoms and functioning over time during or after interventions. Availability of established cut-points for screening and symptom severity, and normative/reference values, may play into choice of PRO measures for use in clinical care. Translatability of thresholds for meaningful change is more problematic because of the greater reliability needed at the individual-level versus group-level, but group-based methods may provide lower bound estimates. Caution is needed to set thresholds above bounds of measurement error to avoid "false-positive changes" triggering unwarranted alerts and action in clinic.
While there are some challenges in applying available methods for interpreting group-based PRO results to individual patient data and clinical care-including myriad contextual factors that may influence an individual patient's management and decision-making-they provide a useful starting point, and should be used pragmatically.
患者报告的结局（PRO）数据可在两个层面上使用：评估疾病和治疗对个体（群体水平）的影响，以及对个体患者进行筛查/监测，以告知其管理（个体水平）。为了使 PRO 数据在这两个层面上都有用，我们需要了解其临床相关性。
阐明历史上为解释基于群体的 PRO 研究结果而开发的方法是否以及如何可以在临床环境中应用，以使来自个体患者的 PRO 数据能够为其临床管理和决策提供信息。
我们首先区分了在群体和个体层面上所需的基于 PRO 的决策。然后，我们总结了已建立的基于群体的解释方法（基于锚定和基于分布的方法），以及最近利用项目校准和定性研究方法的方法。然后，我们评估这些方法在个体患者数据和个体层面决策中的适用性。
基于群体的方法提供了一系列在临床护理中有用的阈值：一些为需要进一步临床评估和/或干预的患者提供筛查阈值，一些为个体症状或功能问题严重程度分类提供阈值，而另一些则为监测症状和功能随时间变化提供阈值在干预期间或之后。筛选和症状严重程度的既定切点以及规范/参考值的可用性可能会影响在临床护理中使用 PRO 措施的选择。有意义变化的阈值的可翻译性更成问题，因为在个体层面上比在群体层面上需要更高的可靠性，但基于群体的方法可能提供下限估计。为了避免“假阳性变化”触发不必要的临床警报和行动，需要谨慎地将阈值设定在测量误差的范围之上。
虽然将解释基于群体的 PRO 结果的现有方法应用于个体患者数据和临床护理存在一些挑战，包括可能影响个体患者管理和决策的无数情境因素，但它们提供了一个有用的起点，并且应该务实应用。
