采用OMERACT流程对抗中性粒细胞胞浆抗体相关血管炎患者报告结局的探索、开发与验证
Exploration, Development, and Validation of Patient-reported Outcomes in Antineutrophil Cytoplasmic Antibody-associated Vasculitis Using the OMERACT Process.
作者信息
Robson Joanna C, Milman Nataliya, Tomasson Gunnar, Dawson Jill, Cronholm Peter F, Kellom Katherine, Shea Judy, Ashdown Susan, Boers Maarten, Boonen Annelies, Casey George C, Farrar John T, Gebhart Don, Krischer Jeffrey, Lanier Georgia, McAlear Carol A, Peck Jacqueline, Sreih Antoine G, Tugwell Peter S, Luqmani Raashid A, Merkel Peter A
机构信息
From the Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Science, University of Oxford, Oxford, UK; Division of Rheumatology, University of Ottawa, Ottawa, Ontario, Canada; Department of Public Health Sciences, University of Iceland, Reykjavik, Iceland; Nuffield Department of Population Health (HSRU), University of Oxford, Oxford, UK; Department of Family Medicine and Community Health, and Division of Rheumatology and Department of Biostatistics and Epidemiology, University of Pennsylvania, and Department of Medicine, Hospital of the University of Pennsylvania, Philadelphia, Pennsylvania, USA; Departments of Epidemiology and Biostatistics; and Rheumatology, VU University Medical Center, Amsterdam, The Netherlands; Division of Rheumatology, Maastricht University Medical Center and Caphri Research Institute, University Maastricht, Maastricht, The Netherlands; Vasculitis Foundation, Denver, Colorado, USA; Division of Biostatistics and Informatics, Department of Pediatrics, College of Medicine, University of South Florida, Tampa, Florida, USA; Vasculitis Clinical Research Consortium, and Division of Rheumatology, University of Pennsylvania, Philadelphia, Pennsylvania, USA.J.C. Robson, MBBS, PhD, Clinical Lecturer in Rheumatology, Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Science, University of Oxford; N. Milman, MD, FRCPC, Clinical Lecturer in Rheumatology, Division of Rheumatology, University of Ottawa; G. Tomasson, MD, Department of Public Health Sciences, University of Iceland; J. Dawson, MA, MSc, DPhil, University Research Lecturer and Senior Research Scientist, Nuffield Department of Population Health (HSRU), University of Oxford; P.F. Cronholm, MD, MSCE, FAAFP, Associate Professor, Department of Family Medicine and Community Health, University of Pennsylvania; K. Kellom, BA, Senior Research Coordinator, Department of Family Medicine and Community Health, University of Pennsylvania; J. Shea, PhD, Professor of Medici
出版信息
J Rheumatol. 2015 Nov;42(11):2204-9. doi: 10.3899/jrheum.141143. Epub 2015 Sep 1.
OBJECTIVE
Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of linked multisystem life- and organ-threatening diseases. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group has been at the forefront of outcome development in the field and has achieved OMERACT endorsement of a core set of outcomes for AAV. Patients with AAV report as important some manifestations of disease not routinely collected through physician-completed outcome tools; and they rate common manifestations differently from investigators. The core set includes the domain of patient-reported outcomes (PRO). However, PRO currently used in clinical trials of AAV do not fully characterize patients' perspectives on their burden of disease. The OMERACT vasculitis working group is addressing the unmet needs for PRO in AAV.
METHODS
Current activities of the working group include (1) evaluating the feasibility and construct validity of instruments within the PROMIS (Patient-Reported Outcome Measurement Information System) to record components of the disease experience among patients with AAV; (2) creating a disease-specific PRO measure for AAV; and (3) applying The International Classification of Functioning, Disability and Health to examine the scope of outcome measures used in AAV.
RESULTS
The working group has developed a comprehensive research strategy, organized an investigative team, included patient research partners, obtained peer-reviewed funding, and is using a considerable research infrastructure to complete these interrelated projects to develop evidence-based validated outcome instruments that meet the OMERACT filter of truth, discrimination, and feasibility.
CONCLUSION
The OMERACT vasculitis working group is on schedule to achieve its goals of developing validated PRO for use in clinical trials of AAV.
目的
抗中性粒细胞胞浆抗体（ANCA）相关血管炎（AAV）是一组危及生命和器官的多系统相关疾病。风湿病结局评估（OMERACT）血管炎工作组一直处于该领域结局指标开发的前沿，并已使AAV的一组核心结局指标获得OMERACT认可。AAV患者报告称，一些疾病表现很重要，但这些表现并非通过医生填写的结局工具常规收集；而且他们对常见表现的评价与研究人员不同。核心指标集包括患者报告结局（PRO）领域。然而，目前AAV临床试验中使用的PRO并不能完全体现患者对疾病负担的看法。OMERACT血管炎工作组正在解决AAV中PRO未得到满足的需求。
方法
工作组目前的活动包括：（1）评估患者报告结局测量信息系统（PROMIS）中的工具记录AAV患者疾病体验组成部分的可行性和结构效度；（2）为AAV创建特定疾病的PRO测量指标；（3）应用《国际功能、残疾和健康分类》来审视AAV中使用的结局指标范围。
结果
工作组制定了全面的研究策略，组建了研究团队，纳入了患者研究伙伴，获得了同行评审资金，并正在利用大量研究基础设施来完成这些相互关联的项目，以开发基于证据的、经过验证的结局工具，使其符合OMERACT关于真实性、区分度和可行性的标准。
结论
OMERACT血管炎工作组按计划实现其目标，即开发经过验证的PRO，用于AAV的临床试验。
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