Helliwell Toby, Brouwer Elisabeth, Pease Colin T, Hughes Rodney, Hill Catherine L, Neill Lorna M, Halls Serena, Simon Lee S, Mallen Christian D, Boers Maarten, Kirwan John R, Mackie Sarah L
From the Department of Primary Care Sciences, Keele University, Staffordshire, UK; Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Center, Groningen, The Netherlands; Department of Rheumatology, Leeds Teaching Hospitals ( UK) National Health Service (NHS) Trust, Leeds, UK; Department of Rheumatology Ashford and St. Peter's Hospitals NHS Foundation Trust, Chertsey, Surrey, UK; Rheumatology Unit, The Queen Elizabeth Hospital, University of Adelaide, Woodville, South Australia; PMR-GCA Scotland, Forest Lodge, Foulden, Berwickshire, UK; University of the West of England, Academic Rheumatology Unit, Bristol Royal Infirmary, Bristol, UK; SDC LLC, Cambridge, Massachusetts, USA; Department of Primary Care Sciences, Keele University, Staffordshire, UK; Departments of Epidemiology and Biostatistics, and Rheumatology, VU University Medical Center, Amsterdam, The Netherlands; University of Bristol, Academic Rheumatology Unit, Bristol Royal Infirmary, Bristol, UK; UK National Institute for Health Research (NIHR)-Leeds Biomedical Research Unit, Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, UK.T. Helliwell, MRCGP, MMedSci, Department of Primary Care Sciences, Keele University; E. Brouwer, MD, PhD, Department of Rheumatology and Clinical Immunology, University of Groningen, University Medical Center; C.T. Pease, MD, FRCP, Department of Rheumatology, Leeds Teaching Hospitals NHS Trust; R. Hughes, MA, MD, FRCP, Department of Rheumatology Ashford and St. Peter's Hospitals NHS Foundation Trust; C.L. Hill, MBBS, MD, MSc, FRACP, Staff Specialist, Rheumatology Unit, Queen Elizabeth Hospital, University of Adelaide; L.M. Neill, BSc, CPhys, MInstP, Trustee and Secretary, PMR-GCA Scotland; S. Halls, MSc, BSc, University of the West of England, Academic Rheumatology Unit, Bristol Royal Infirmary; L.S. Simon, MD, SDC LLC; C.D. Mallen, BMBS, PhD, FRCGP, Department of Primary Care Sciences, Keele University;
J Rheumatol. 2016 Jan;43(1):182-6. doi: 10.3899/jrheum.141179. Epub 2015 Nov 15.
The Outcome Measures in Rheumatology (OMERACT) polymyalgia rheumatica (PMR) working group aims to develop a core set of outcome measures to be used in clinical trials for PMR. Previous reports from OMERACT 11 included a qualitative study of the patient experience and a preliminary literature review.
A 3-round Delphi survey of clinicians and patients with PMR was undertaken to identify a candidate core domain set for PMR research. Additionally, a literature review of outcome measures and their respective measurement instruments was undertaken. Meetings of patient research partners and clinicians were convened to review face validity of the provisional core domain set, which was subsequently presented and discussed at the OMERACT 12 congress.
Of the 60 clinicians taking part in round 1, 55 took part in round 2 and 51 in round 3. Of the 55 patients who took part in round 1, 46 and 35 took part in subsequent rounds. In total, 91% of participants in round 3 deemed the resulting draft core domain set reasonable. The literature review identified 28 studies for full review. Measurement instruments for each proposed domain were identified. Clinicians are highly aware of glucocorticoid-related adverse effects, but there is relatively little evidence about their true prevalence and severity, especially in PMR.
A provisional core domain set, presented for clinical trials in PMR, comprises acute phase markers, physical function, death, glucocorticoid-related adverse events, and development of giant cell arteritis. Measurement instruments are suggested that may cover each domain, but these require formal validation for clinical trials in PMR.
风湿病疗效评估（OMERACT）多肌痛（PMR）工作组旨在制定一套核心疗效评估指标，用于PMR的临床试验。OMERACT 11的先前报告包括对患者体验的定性研究和初步文献综述。
对PMR临床医生和患者进行三轮德尔菲调查，以确定PMR研究的候选核心领域集。此外，对疗效评估指标及其各自的测量工具进行了文献综述。召开了患者研究伙伴和临床医生会议，以审查临时核心领域集的表面效度，该领域集随后在OMERACT 12大会上进行了展示和讨论。
参与第一轮的60名临床医生中，55名参与了第二轮，51名参与了第三轮。参与第一轮的55名患者中，46名和35名参与了后续轮次。总共91%的第三轮参与者认为最终的核心领域集草案合理。文献综述确定了28项研究进行全面审查。确定了每个提议领域的测量工具。临床医生高度意识到糖皮质激素相关的不良反应，但关于其真实患病率和严重程度的证据相对较少，尤其是在PMR中。
提出了一个用于PMR临床试验的临时核心领域集，包括急性期标志物、身体功能、死亡、糖皮质激素相关不良事件和巨细胞动脉炎的发生。建议了可能涵盖每个领域的测量工具，但这些工具需要在PMR临床试验中进行正式验证。
