Varian Frances, Burney Rebecca, Pearson Charlotte, Goh Ze Ming, Newman Joseph, Rawlings Gregg, Zafar Hamza, Kiely David G, Thompson A A Roger, Condliffe Robin, Toshner Mark, McCormack Ciara, Armstrong Iain, Peasgood Tessa, Carlton Jill, Rothman Alexander M K
Division of Clinical Medicine, School of Medicine and Population Health (formerly Faculty of Medicine, Dentistry and Health), University of Sheffield, Sheffield, UK.
Sheffield Pulmonary Vascular Disease Unit, Royal Hallamshire Hospital, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK.
Eur Respir Rev. 2025 May 14;34(176). doi: 10.1183/16000617.0006-2025. Print 2025 Apr.
Health-related quality of life (HRQoL) in pulmonary arterial hypertension (PAH) is valued as an outcome measure by patients, clinicians and regulators. The selection of patient-reported outcome measures (PROMs) for measurement of HRQoL in PAH clinical trials lacks systematic evaluation of their suitability, accuracy and reliability.
We report a systematic review (PROSPERO ID: CRD42024484021) following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines of PROMs selected in PAH clinical trials. PROM measurement properties were then evaluated according to the 10-step COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist and graded by recommendation for use. Finally, HRQoL was modelled into a conceptual framework using patient interviews and surveys.
Screening of 896 records identified 90 randomised controlled trials. 43 trials selected PROMs, of which 20 were sufficiently validated to detect meaningful change. Of these, eight trials were adequately powered, using either EuroQol-five dimensions-five levels (EQ-5D-5L), Short-Form-36 (SF-36) or the Living with Pulmonary Hypertension Questionnaire (LPHQ). The COSMIN evaluation recommended EmPHasis-10 and the LPHQ for use (grade A); whereas, SF-36 and EQ-5D-5L require further study (grade B). A conceptual framework of HRQoL was developed from literature comprising 8045 patients. This framework can be used to visualise the different HRQoL concepts measured by different PROMs.
To improve patient-centred research, greater consistency in PROM selection is required. Three of 90 randomised controlled trials have selected COSMIN-recommended PROMs. Whilst the PROMs evaluated require development across the 10 areas of psychometric property measurement, EmPHasis-10 and the LPHQ can be recommended for use. The ratified conceptual framework can further support PROM selection by identifying the HRQoL concepts they are likely to capture.
肺动脉高压（PAH）患者的健康相关生活质量（HRQoL）被患者、临床医生和监管机构视为一项重要的疗效指标。在PAH临床试验中，用于测量HRQoL的患者报告结局指标（PROMs）的选择缺乏对其适用性、准确性和可靠性的系统评估。
我们按照系统评价和Meta分析的首选报告项目（PRISMA）指南，对PAH临床试验中选择的PROMs进行了一项系统评价（PROSPERO注册号：CRD42024484021）。然后根据基于共识的健康测量工具选择标准（COSMIN）清单的10个步骤对PROM测量属性进行评估，并根据使用建议进行分级。最后，通过患者访谈和调查将HRQoL纳入一个概念框架。
对896条记录进行筛选后，确定了90项随机对照试验。43项试验选择了PROMs，其中20项经过充分验证，能够检测出有意义的变化。其中，八项试验样本量充足，使用了欧洲五维度五水平健康量表（EQ-5D-5L）、简短健康调查问卷（SF-36）或肺动脉高压生活问卷（LPHQ）。COSMIN评估推荐使用EmPHasis-10和LPHQ（A级）；而SF-36和EQ-5D-5L需要进一步研究（B级）。从包含8045名患者的文献中建立了一个HRQoL概念框架。该框架可用于直观展示不同PROMs所测量的不同HRQoL概念。
为了改进以患者为中心的研究，需要在PROM选择上有更高的一致性。90项随机对照试验中有三项选择了COSMIN推荐的PROMs。虽然所评估的PROMs需要在心理测量属性测量的10个领域进行改进，但可以推荐使用EmPHasis-10和LPHQ。经过验证的概念框架可以通过识别它们可能捕捉到的HRQoL概念，进一步支持PROM的选择。
