Choy Ernest H, Khoshaba Bernadette, Cooper Derek, MacGregor Alex, Scott David L
Sir Alfred Baring Garrod Clinical Trials Unit, Academic Department of Rheumatology, King's College London, London, UK.
Arthritis Rheum. 2008 Feb 15;59(2):192-9. doi: 10.1002/art.23342.
Assessor-based disease activity measures such as the Disease Activity Score in 28 joints (DAS28), although widely used in rheumatoid arthritis (RA), have high interobserver variability. We developed and validated a patient-based disease activity score (PDAS) as an alternative assessment.
Patients' assessments of swollen or tender joints, visual analog scales for pain and general health, the Health Assessment Questionnaire, and erythrocyte sedimentation rate (ESR) were used to develop the PDAS. In a developmental cohort (204 patients), regression analyses determined the best fit with the DAS28. A validation cohort (322 patients) subsequently evaluated criterion and construct validity against a range of outcome measures, including the Nottingham Health Profile (NHP) and Short Form 36 (SF-36). Sensitivity to change was assessed in 56 patients after 6 months of treatment with disease-modifying antirheumatic drugs or biologics.
In the developmental cohort, the PDAS with ESR (PDAS1) and without ESR (PDAS2) achieved excellent fit with the DAS28 (r = 0.88 and 0.74, respectively). In the validation cohort, the PDAS showed high criterion validity by correlation with the DAS28 (PDAS1: r = 0.89, PDAS2: r = 0.76). Construct validity was demonstrated by high correlations with a range of disease activity measures (r > or = 0.45), whereas low correlations (r < 0.45) with mental and social components of the SF-36 and NHP indicated divergent validity. The PDAS and DAS28 had similar sensitivity to change, determined using effect sizes (DAS28 = 1.03, PDAS1 = 1.02, PDAS2 = 0.77) or standardized response means (DAS28 = 0.79, PDAS1 = 0.77, PDAS2 = 0.73).
The PDAS1 and PDAS2 are valid and sensitive tools to assess disease activity in RA. They appear suitable for clinical decision making, epidemiologic research, and clinical trials.
基于评估者的疾病活动度测量方法，如28个关节疾病活动评分（DAS28），虽在类风湿关节炎（RA）中广泛应用，但观察者间变异度较高。我们开发并验证了一种基于患者的疾病活动度评分（PDAS）作为替代评估方法。
采用患者对肿胀或压痛关节的评估、疼痛和总体健康状况的视觉模拟量表、健康评估问卷以及红细胞沉降率（ESR）来制定PDAS。在一个开发队列（204例患者）中，通过回归分析确定与DAS28的最佳拟合度。随后，一个验证队列（322例患者）针对一系列结局指标，包括诺丁汉健康量表（NHP）和简明健康调查问卷36项版（SF - 36），评估了标准效度和结构效度。在56例接受改善病情抗风湿药物或生物制剂治疗6个月后的患者中评估了对变化的敏感性。
在开发队列中，含ESR的PDAS（PDAS1）和不含ESR的PDAS（PDAS2）与DAS28的拟合度均极佳（分别为r = 0.88和0.74）。在验证队列中，PDAS与DAS28的相关性显示出较高的标准效度（PDAS1：r = 0.89，PDAS2：r = 0.76）。与一系列疾病活动度测量指标的高相关性证明了结构效度（r≥0.45），而与SF - 36和NHP的心理及社会成分的低相关性（r < 0.45）则表明了区分效度。使用效应量（DAS28 = 1.03，PDAS1 = 1.02，PDAS2 = 0.77）或标准化反应均值（DAS28 = 0.79，PDAS1 = 0.77，PDAS2 = 0.73）确定，PDAS和DAS28对变化的敏感性相似。
PDAS1和PDAS2是评估RA疾病活动度有效且敏感的工具。它们似乎适用于临床决策、流行病学研究和临床试验。
