Walter Margot J, Mohd Din S H, Hazes Johanna M, Lesaffre E, Barendregt P J, Luime Jolanda J
From the Department of Rheumatology and the Department of Biostatistics, University Medical Center; Department of Rheumatology, Maasstad Hospital, Rotterdam, the Netherlands; and Department of Biostatistics, KV Leuven, Leuven, Belgium.
J Rheumatol. 2014 Apr;41(4):640-7. doi: 10.3899/jrheum.130174. Epub 2014 Feb 15.
To evaluate the performance of patient-reported outcomes (PRO) as primary indices for identification and prediction of a 28-joint Disease Activity Score (DAS28)>3.2 among patients with rheumatoid arthritis (RA).
Patients with RA completed monthly online PRO [Health Assessment Questionnaire (HAQ), Rheumatoid Arthritis Disease Activity Index (RADAI), visual analog scale (VAS) fatigue] and were clinically assessed every 3 months using the DAS28. Simple descriptive statistics, logistic regression, and the Bayesian joint modeling approach were used to analyze the data. The Bayesian joint model combines the scores and changes in the scores of 3 PRO to predict a DAS28>3.2 at the subsequent timepoint.
A group of 159 patients with RA participated. Stratified summaries of the PRO by DAS28 categories at baseline provided incremental values of the PRO for more active disease. However, on an individual level, the DAS28 and the PRO fluctuated over time. The prediction of subsequent DAS score by a single instrument at single timepoints resulted in moderate sensitivity and specificity. Using the intercept and slope of the combined PRO of the first 3 measurements to predict the DAS28 state at 3 months resulted in a sensitivity of 0.81 and a specificity of 0.92. After 10-fold cross validation, the model had a sensitivity of 0.61 and specificity of 0.75 to identify patients with a DAS28>3.2.
PRO showed fluctuating levels of disease activity over time, while on a group level disease activity stayed the same. Using the changes in RADAI, HAQ, and VAS fatigue over time to predict future DAS28>3.2 resulted in moderate performance after the internal cross-validation of the model (sensitivity 0.61, specificity 0.75).
评估患者报告结局（PRO）作为类风湿关节炎（RA）患者中识别和预测28关节疾病活动评分（DAS28）>3.2的主要指标的性能。
RA患者每月完成在线PRO[健康评估问卷（HAQ）、类风湿关节炎疾病活动指数（RADAI）、视觉模拟量表（VAS）疲劳]，并每3个月使用DAS28进行临床评估。使用简单描述性统计、逻辑回归和贝叶斯联合建模方法分析数据。贝叶斯联合模型结合3个PRO的评分和评分变化，以预测后续时间点的DAS28>3.2。
159例RA患者参与研究。基线时按DAS28类别对PRO进行分层总结，为疾病活动度更高的患者提供了PRO的增量值。然而，在个体水平上，DAS28和PRO随时间波动。在单个时间点使用单一工具预测后续DAS评分，其敏感性和特异性中等。使用前3次测量的联合PRO的截距和斜率预测3个月时的DAS28状态，敏感性为0.81，特异性为0.92。经过10倍交叉验证后，该模型识别DAS28>3.2患者的敏感性为0.61，特异性为0.75。
PRO显示疾病活动度随时间波动，而在群体水平上疾病活动度保持不变。使用RADAI、HAQ和VAS疲劳随时间的变化来预测未来DAS28>3.2，在模型内部交叉验证后性能中等（敏感性0.61，特异性0.75）。
