风湿病学中的适应性试验设计：来自 OMERACT 特别兴趣小组的报告。
Adaptive Trial Designs in Rheumatology: Report from the OMERACT Special Interest Group.
机构信息
From the CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University, Cardiff, UK; Schlosspark Klinik, Charité University Medicine, Berlin, Germany; Department of Epidemiology and Biostatistics; Amsterdam Rheumatology and Immunology Center; Amsterdam University Medical Centers, location VUmc, Amsterdam, the Netherlands; Department of Rheumatology, Hospital for Special Surgery, Weill Cornell Medical College; Pfizer, New York, New York; Sanofi, Bridgewater, New Jersey; SDG LLC, Cambridge, Massachusetts, USA; Rebecca McDonald Centre for Arthritis and Autoimmune Disease, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada; Musculoskeletal Statistics Unit, The Parker Institute, Bispebjerg and Frederiksberg Hospital; Department of Rheumatology, Odense University Hospital, Odense, Denmark; Division of Rheumatology, Department of Medicine and Therapeutics, and Faculty of Medicine, The Chinese University of Hong Kong, Hong Kong.
T. Pickles, MSc, CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University; R. Alten, MD, PhD, Schlosspark Klinik, Charité University Medicine; M. Boers, MD, PhD, MSc, Department of Epidemiology and Biostatistics, and Amsterdam Rheumatology and Immunology Center, and Amsterdam University Medical Centers VUmc; V. Bykerk, MD, FRCPC, Department of Rheumatology, Hospital for Special Surgery, Weill Cornell Medical College, and Rebecca McDonald Centre for Arthritis and Autoimmune Disease, Mount Sinai Hospital, University of Toronto; J. Christensen, MD, Pfizer; R. Christensen, PhD, MSc, Musculoskeletal Statistics Unit, The Parker Institute, Bispebjerg and Frederiksberg Hospital, and Department of Rheumatology, Odense University Hospital; H. van Hoogstraten, MD, PhD, Sanofi; L.S. Simon, MD, SDG LLC; L.S. Tam, MD, Division of Rheumatology, Department of Medicine and Therapeutics, and Faculty of Medicine, The Chinese University of Hong Kong; E.H. Choy, MD, CREATE Centre, Section of Rheumatology, Division of Infection and Immunity, Cardiff University.
出版信息
J Rheumatol. 2019 Oct;46(10):1406-1408. doi: 10.3899/jrheum.181054. Epub 2019 Feb 15.
OBJECTIVE
Adaptive trial design was developed initially for oncology to improve trial efficiency. If optimized for rheumatology, it may improve trial efficiency by reducing sample size and time.
METHODS
A systematic review assessed design of phase II clinical trials in rheumatoid arthritis.
RESULTS
Fifty-six trials were reviewed. Most trials had 4 groups (1 control and 3 intervention), with an average group size of 34 patients. American College of Rheumatology 20 measured at 16 weeks was the most commonly used primary endpoint.
CONCLUSION
The next step is to undertake a systematic review of adaptive designs used in early-phase trials in nonrheumatic conditions.
目的
适应性试验设计最初是为肿瘤学开发的，以提高试验效率。如果针对风湿病进行优化，它可以通过减少样本量和时间来提高试验效率。
方法
系统评价评估了类风湿关节炎的 II 期临床试验设计。
结果
共审查了 56 项试验。大多数试验有 4 个组（1 个对照组和 3 个干预组），每组平均 34 名患者。最常用于主要终点的是在 16 周时测量的美国风湿病学会 20 分。
结论
下一步是对非风湿性疾病早期试验中使用的适应性设计进行系统评价。
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