Frances Varian, Rebecca Burney, Charlotte Pearson, Ze Ming Goh, Joseph Newman, Gregg Rawlings, Hamza Zafar, David G Kiely, A A Roger Thompson, Robin Condliffe, Mark Toshner, Ciara McCormack, Iain Armstrong, Tessa Peasgood, Jill Carlton, Alexander M K Rothman
{"title":"肺动脉高压临床试验中患者报告的结局指标的选择：系统回顾、荟萃分析和健康相关生活质量框架","authors":"Frances Varian, Rebecca Burney, Charlotte Pearson, Ze Ming Goh, Joseph Newman, Gregg Rawlings, Hamza Zafar, David G Kiely, A A Roger Thompson, Robin Condliffe, Mark Toshner, Ciara McCormack, Iain Armstrong, Tessa Peasgood, Jill Carlton, Alexander M K Rothman","doi":"10.1183/16000617.0006-2025","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":12166,"journal":{"name":"European Respiratory Review","volume":"34 176","pages":""},"PeriodicalIF":9.0000,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12076161/pdf/","citationCount":"0","resultStr":"{\"title\":\"Selection of patient-reported outcome measures in pulmonary arterial hypertension clinical trials: a systematic review, meta-analysis and health-related quality of life framework.\",\"authors\":\"Frances Varian, Rebecca Burney, Charlotte Pearson, Ze Ming Goh, Joseph Newman, Gregg Rawlings, Hamza Zafar, David G Kiely, A A Roger Thompson, Robin Condliffe, Mark Toshner, Ciara McCormack, Iain Armstrong, Tessa Peasgood, Jill Carlton, Alexander M K Rothman\",\"doi\":\"10.1183/16000617.0006-2025\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p>\",\"PeriodicalId\":12166,\"journal\":{\"name\":\"European Respiratory Review\",\"volume\":\"34 176\",\"pages\":\"\"},\"PeriodicalIF\":9.0000,\"publicationDate\":\"2025-05-14\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12076161/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"European Respiratory Review\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1183/16000617.0006-2025\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/4/1 0:00:00\",\"PubModel\":\"Print\",\"JCR\":\"Q1\",\"JCRName\":\"RESPIRATORY SYSTEM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"European Respiratory Review","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1183/16000617.0006-2025","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/4/1 0:00:00","PubModel":"Print","JCR":"Q1","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
Selection of patient-reported outcome measures in pulmonary arterial hypertension clinical trials: a systematic review, meta-analysis and health-related quality of life framework.
Introduction: 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.
Methods: 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.
Results: 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.
Conclusion: 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.
期刊介绍：
The European Respiratory Review (ERR) is an open-access journal published by the European Respiratory Society (ERS), serving as a vital resource for respiratory professionals by delivering updates on medicine, science, and surgery in the field. ERR features state-of-the-art review articles, editorials, correspondence, and summaries of recent research findings and studies covering a wide range of topics including COPD, asthma, pulmonary hypertension, interstitial lung disease, lung cancer, tuberculosis, and pulmonary infections. Articles are published continuously and compiled into quarterly issues within a single annual volume.
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