AMERICAN-COLLEGE-OF-RHEUMATOLOGY PRELIMINARY DEFINITION OF IMPROVEMENT IN RHEUMATOID-ARTHRITIS
AMERICAN-COLLEGE-OF-RHEUMATOLOGY PRELIMINARY DEFINITION OF IMPROVEMENT IN RHEUMATOID-ARTHRITIS
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DOI：
10.1002/art.1780380602
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发表时间：
1995-06-01
影响因子：
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通讯作者：
KIESZAK, S
中科院分区：
文献类型：
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作者：
FELSON, DT;ANDERSON, JJ;KIESZAK, S
目的：类风湿关节炎（RA）治疗的试验报告了治疗患者在多个结果测量中的平均反应。检验个体患者是否在治疗后得到改善更具有临床意义，这确定了一个单一的主要疗效指标。目前在不同的试验中使用了多种改善的定义，本研究的目的是颁布一个用于RA试验的单一定义。使用美国风湿病学会（ACR） RA试验的核心结果测量标准，我们使用三步流程测试了40种不同的改善定义。首先，我们对风湿病学家进行了调查，使用来自试验的实际患者病例，评估哪些定义最符合风湿病学家对改善的印象，消除大多数候选的改善定义。其次，我们测试了剩余的20个定义，以确定哪些定义最大程度地区别于安慰剂治疗的有效治疗，并将安慰剂反应率降至最低。在剩下的8个候选改善定义中，我们测试了哪些定义最容易使用，哪些定义最符合风湿病学家对改善的印象。选择以下改善定义：压痛和肿胀关节计数改善20%，剩余5项ACR核心组测量中的3项改善20%：患者和医生总体评估、疼痛、残疾和急性期反应物。在一项比较试验中对这一定义进行了进一步的验证，结果表明，该定义在统计上是强有力的，并没有确定很大比例的安慰剂治疗患者得到了改善。我们提出了一个改善的定义，我们希望将广泛用于RA试验，
Objective, Trials of rheumatoid arthritis (RA) treatments report the average response in multiple outcome measures for treated patients. It is more clinically relevant to test whether individual patients improve with treatment, and this identifies a single primary efficacy measure, Multiple definitions of improvement are currently in use in different trials, The goal of this study was to promulgate a single definition for use in RA trials.Methods. Using the American College of Rheumatology (ACR) core set of outcome measures for RA trials, we tested 40 different definitions of improvement, using a 3-step process. First, we performed a survey of rheumatologists, using actual patient cases from trials, to evaluate which definitions corresponded best to rheumatologists' impressions of improvement, eliminating most candidate definitions of improvement. Second, we tested 20 remaining definitions to determine which maximally discriminated effective treatment from placebo treatment and also minimized placebo response rates, With 8 candidate definitions of improvement remaining, we tested to see which were easiest to use and were best in accord with rheumatologists' impressions of improvement,Results. The following definition of improvement was selected: 20% improvement in tender and swollen joint counts and 20% improvement in 3 of the 5 remaining ACR core set measures: patient and physician global assessments, pain, disability, and an acute-phase reactant. Additional validation of this definition was carried out in a comparative trial, and the results suggest that the definition is statistically powerful and does not identify a large percentage of placebo-treated patients as being improved.Conclusion. We present a definition of improvement which we hope will be used widely in RA trials,