Amsterdam UMC, Department of Epidemiology and Data Science, Vrije Universiteit Amsterdam, Amsterdam, the Netherlands.
Amsterdam Public Health Research Institute, Methodology, Amsterdam, the Netherlands.
Diabetologia. 2023 Aug;66(8):1357-1377. doi: 10.1007/s00125-023-05926-3. Epub 2023 May 24.
Patient-reported outcomes (PROs) are valuable for shared decision making and research. Patient-reported outcome measures (PROMs) are questionnaires used to measure PROs, such as health-related quality of life (HRQL). Although core outcome sets for trials and clinical practice have been developed separately, they, as well as other initiatives, recommend different PROs and PROMs. In research and clinical practice, different PROMs are used (some generic, some disease-specific), which measure many different things. This is a threat to the validity of research and clinical findings in the field of diabetes. In this narrative review, we aim to provide recommendations for the selection of relevant PROs and psychometrically sound PROMs for people with diabetes for use in clinical practice and research. Based on a general conceptual framework of PROs, we suggest that relevant PROs to measure in people with diabetes are: disease-specific symptoms (e.g. worries about hypoglycaemia and diabetes distress), general symptoms (e.g. fatigue and depression), functional status, general health perceptions and overall quality of life. Generic PROMs such as the 36-Item Short Form Health Survey (SF-36), WHO Disability Assessment Schedule (WHODAS 2.0), or Patient-Reported Outcomes Measurement Information System (PROMIS) measures could be considered to measure commonly relevant PROs, supplemented with disease-specific PROMs where needed. However, none of the existing diabetes-specific PROM scales has been sufficiently validated, although the Diabetes Symptom Self-Care Inventory (DSSCI) for measuring diabetes-specific symptoms and the Diabetes Distress Scale (DDS) and Problem Areas in Diabetes (PAID) for measuring distress showed sufficient content validity. Standardisation and use of relevant PROs and psychometrically sound PROMs can help inform people with diabetes about the expected course of disease and treatment, for shared decision making, to monitor outcomes and to improve healthcare. We recommend further validation studies of diabetes-specific PROMs that have sufficient content validity for measuring disease-specific symptoms and consider generic item banks developed based on item response theory for measuring commonly relevant PROs.
患者报告的结局（PROs）对于共同决策和研究非常有价值。患者报告结局测量（PROMs）是用于测量 PROs 的问卷，例如健康相关生活质量（HRQL）。尽管已经分别为试验和临床实践制定了核心结局集，但它们以及其他倡议建议使用不同的 PROs 和 PROMs。在研究和临床实践中，使用了不同的 PROM（有些是通用的，有些是特定于疾病的），这些 PROM 测量了许多不同的内容。这对糖尿病领域的研究和临床发现的有效性构成了威胁。在这篇叙述性综述中，我们旨在为糖尿病患者选择相关的 PROs 和心理测量上可靠的 PROM 提供建议，以便在临床实践和研究中使用。基于 PROs 的一般概念框架，我们建议在糖尿病患者中测量的相关 PROs 是：特定于疾病的症状（例如对低血糖和糖尿病困扰的担忧）、一般症状（例如疲劳和抑郁）、功能状态、一般健康感知和整体生活质量。通用的 PROM ，如 36 项简短健康调查（SF-36）、世界卫生组织残疾评估量表（WHODAS 2.0）或患者报告的结局测量信息系统（PROMIS）测量，可以考虑用于测量常见的相关 PROs，并在需要时补充特定于疾病的 PROM。然而，尽管用于测量特定于疾病的症状的糖尿病症状自我护理量表（DSSCI）和用于测量困扰的糖尿病困扰量表（DDS）和糖尿病问题领域量表（PAID）具有足够的内容效度，但目前尚无足够的验证过的特定于糖尿病的 PROM 量表。标准化和使用相关的 PROs 和心理测量上可靠的 PROMs 可以帮助糖尿病患者了解疾病和治疗的预期过程，进行共同决策，监测结果并改善医疗保健。我们建议进一步验证具有足够内容效度的用于测量特定于疾病的症状的糖尿病特定 PROM 的研究，并考虑使用基于项目反应理论开发的通用项目库来测量常见的相关 PROs。
