建立一个更新的幼年特发性关节炎研究核心领域集：来自 OMERACT 2018 JIA 研讨会的报告。
Establishing an Updated Core Domain Set for Studies in Juvenile Idiopathic Arthritis: A Report from the OMERACT 2018 JIA Workshop.
机构信息
From the Division of Rheumatology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio; Office of Research, Division of Rheumatology, Columbia University Medical Center, New York; Division of Rheumatology, University of Rochester, Golisano Children's Hospital, Rochester, New York; Division of Rheumatology, Johns Hopkins University, Baltimore, Maryland; Thornhill Associates, Hermosa Beach, California; Division of Rheumatology, Children's Mercy, Kansas City, Kansas City, Missouri; Division of Pediatric Rheumatology, Rutgers Robert Wood Johnson Medical School, Institute for Health, Health Care Policy and Aging Research, New Brunswick, New Jersey; University of Alabama at Birmingham, Birmingham, Alabama; Division of Rheumatology, Seattle Children's Hospital, Seattle, Washington; Patient Engagement, Arthritis Foundation, Atlanta, Georgia; University of Florida, Shands Children's Hospital, Gainesville, Florida; Division of Rheumatology, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; USA; Royal Children's Hospital; Murdoch Children's Research Institute Melbourne, Melbourne; Consumer and Community Health Research Network, Crawley, Australia; Ottawa Hospital Research Institute; School of Epidemiology and Public Health, University of Ottawa; Department of Pediatrics and School of Rehabilitation Sciences, University of Ottawa; Children's Hospital of Eastern Ontario Research Institute, Ottawa; Institute of Health Policy Management and Evaluation, University of Toronto; Division of Rheumatology, The Hospital for Sick Children; Child Health Evaluative Sciences, Hospital for Sick Children, Toronto, Ontario, Canada; Institute for Research and Health Care (IRCCS) Istituto Giannina Gaslini, Clinica Pediatrica e Reumatologia; Università degli studi di Genova, Genoa; Rheumatology Division, Ospedale Pediatrico Bambino Gesù, Rome, Italy; Amsterdam Rheumatology and Immunology Center/Reade l Emma Children's Hospital, Amsterdam Medical Center, Amsterdam, the Netherlands; Rheumatology, Respiratory, Gastroenterology and Immunology Office Scientific and Regulatory Management Department European Medicines Agency, London, UK; University of Nis, Faculty of Medicine, Department of Pediatric Rheumatology, Nis, Serbia; Pediatric Rheumatology Research Institute, Bad Bramstedt, Germany.
E.M. Morgan, MD, Associate Professor, Division of Rheumatology, Cincinnati Children's Hospital Medical Center, University of Cincinnati; J.E. Munro, MBBS, Associate Professor, Head of Rheumatology at the Royal Children's Hospital, and Group Leader, Arthritis research, Murdoch Children's Research Institute; J. Horonjeff, PhD, Instructor, Office of Research, Division of Rheumatology, Columbia University Medical Center; B. Horgan, Consumer Advocate, Consumer and Community Health Research Network; B. Shea, PhD, Clinical Investigator, Ottawa Hospital Research Institute, and Adjunct Professor, School of Epidemiology and Public Health, University of Ottawa; B.M. Feldman, MD, MSc, FRCPC, Professor, Pediatrics, Medicine, Institute of Health Policy Management and Evaluation, University of Toronto, and Head, Division of Rheumatology, The Hospital for Sick Children; H. Clairman, MSc, Clinical Research Project Coordinator, Child Health Evaluative Sciences, Hospital for Sick Children; C.O. Bingham III, MD, Professor of Medicine, Division of Rheumatology, Johns Hopkins University; S. Thornhill, AA, Qualitative Research Consultant, Thornhill Associates; V. Strand, MD, Biopharmaceutical Consultant; A. Alongi, PhD Student, IRCCS Istituto Giannina Gaslini, Clinica Pediatrica e Reumatologia, and Università degli studi di Genova; S. Magni-Manzoni, MD, Rheumatology Division, Ospedale Pediatrico Bambino Gesù; M.A. van Rossum, MD, PhD, Pediatric Rheumatologist/Immunologist, Amsterdam Rheumatology and Immunology Center/Reade l Emma Children's Hospital, Amsterdam Medical Center; R. Vesely, MD, Head of the Rheumatology, Respiratory, Gastroenterology and Immunology Office Scientific and Regulatory Management Department, European Medicines Agency; J. Vojinovic, MD, PhD, Professor, University of Nis, Faculty of Medicine, Department of Pediatric Rheumatology; H.I. Brunner, Professor, Division of Rheumatology, Cincinnati Children's Hospital Medical Center; J.G. Harris, MD, Assistant Professor, Division of Rheumatology, Children's Mercy; D.B. Horton, MD, MSCE, Assistant Professor, Division of Pediatric Rheumatology, Rutgers Robert Wood Johnson Medical School, Institute for Health, Health Care Policy and Aging Research; D.J. Lovell, MD, Professor, Division of Rheumatology, Cincinnati Children's Hospital Medical Center; M. Mannion, MD, Assistant Professor of Rheumatology, University of Alabama at Birmingham; H. Rahimi, MD, Assistant Professor, Division of Rheumatology, University of Rochester, Golisano Children's Hospital; A. Ravelli, MD, Professor of Pediatrics, Università degli Studi di Genova and IRCCS Istituto Giannina Gaslini; S. Ringold, MD, Assistant Professor, Division of Rheumatology, Seattle Children's Hospital; N. Ruperto, MD, MPH, IRCCS Istituto Giannina Gaslini, Clinica Pediatrica e Reumatologia, PRINTO; M.S. Schrandt, JD, Director, Patient Engagement, Arthritis Foundation; S. Shenoi, MBBS, MS Associate Professor, Seattle Children's Hospital; N.J. Shiff, MD, University of Florida, Shands Children's Hospital; K. Toupin-April, PhD, Associate Scientist, Children's Hospital of Eastern Ontario Research Institute, and Assistant Professor, Department of Pediatrics and School of Rehabilitation Sciences, University of Ottawa; N. Tzaribachev, MD, Pediatric Rheumatology Research Institute; P. Weiss, MD, MSCE, Associate Professor, Division of Rheumatology, Children's Hospital of Philadelphia; A. Consolaro, MD, PhD, Assistant Professor, IRCCS Istituto Giannina Gaslini, Clinica Pediatrica e Reumatologia, and Università degli studi di Genova.
出版信息
J Rheumatol. 2019 Aug;46(8):1006-1013. doi: 10.3899/jrheum.181088. Epub 2019 Feb 15.
OBJECTIVE
The current Juvenile Idiopathic Arthritis (JIA) Core Set used in randomized controlled trials (RCT) and longitudinal observational studies (LOS) was developed without the input of patients/parents. At the Outcome Measures in Rheumatology (OMERACT) 2016, a special interest group voted to reconsider the core set, incorporating broader input. We describe subsequent work culminating in an OMERACT 2018 plenary and consensus voting.
METHODS
Candidate domains were identified through literature review, qualitative surveys, and online discussion boards (ODB) held with patients with JIA and parents in Australia, Italy, and the United States. A Delphi process with parents, patients, healthcare providers, researchers, and regulators served to edit the domain list and prioritize candidate domains. After the presentation of results, OMERACT workshop participants voted, with consensus set at > 70%.
RESULTS
Participants in ODB were 53 patients with JIA (ages 15-24 yrs) and 55 parents. Three rounds of Delphi considering 27 domains were completed by 190 (response rate 85%), 201 (84%), and 182 (77%) people, respectively, from 50 countries. There was discordance noted between domains prioritized by patients/parents compared to others. OMERACT conference voting approved domains for JIA RCT and LOS with 83% endorsement. Mandatory domains are pain, joint inflammatory signs, activity limitation/physical function, patient's perception of disease activity (overall well-being), and adverse events. Mandatory in specific circumstances: inflammation/other features relevant to specific JIA categories.
CONCLUSION
Following the OMERACT methodology, we developed an updated JIA Core Domain Set. Next steps are to identify and systematically evaluate best outcome measures for these domains.
目的
目前用于随机对照试验（RCT）和纵向观察研究（LOS）的青少年特发性关节炎（JIA）核心集是在没有患者/家长参与的情况下开发的。在 2016 年的风湿病结局测量（OMERACT）会议上，一个特别兴趣小组投票决定重新考虑核心集，纳入更广泛的意见。我们描述了随后的工作，最终在 2018 年 OMERACT 全体会议上进行了共识投票。
方法
通过文献回顾、定性调查和与澳大利亚、意大利和美国的 JIA 患者和家长进行在线讨论板（ODB），确定候选领域。患者、家长、医疗保健提供者、研究人员和监管机构的德尔菲流程用于编辑领域列表并确定候选领域的优先级。在结果展示后，OMERACT 研讨会参与者进行了投票，共识设定为>70%。
结果
ODB 的参与者包括 53 名 JIA 患者（年龄 15-24 岁）和 55 名家长。经过三轮考虑 27 个领域的 Delphi，分别有来自 50 个国家的 190 人（响应率 85%）、201 人（84%）和 182 人（77%）完成。患者/家长优先考虑的领域与其他人不同。OMERACT 会议投票批准了 JIA RCT 和 LOS 的领域，支持率为 83%。强制性领域包括疼痛、关节炎症迹象、活动受限/身体功能、患者对疾病活动的感知（整体健康状况）和不良事件。在特定情况下强制性：与特定 JIA 类别相关的炎症/其他特征。
结论
按照 OMERACT 方法，我们制定了一个更新的 JIA 核心领域集。下一步是确定和系统地评估这些领域的最佳结局测量方法。
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