Harel Daphna, Hudson Marie, Iliescu Alexandra, Baron Murray, Steele Russell
From the Center for the Promotion of Research Involving Innovative Statistical Methodology (PRIISM) Applied Statistics Center, New York University; Department of Humanities and Social Sciences in the Professions, New York University, New York, New York, USA; Division of Rheumatology, Jewish General Hospital; Lady Davis Institute, Jewish General Hospital; Department of Medicine, McGill University; Mathematics and Statistics, McGill University, Montreal, Quebec, Canada.D. Harel, PhD, PRIISM Applied Statistics Center, New York University, and the Department of Humanities and Social Sciences in the Professions, New York University; M. Hudson, MD, MPH, Division of Rheumatology, Jewish General Hospital, and the Lady Davis Institute, Jewish General Hospital, and the Department of Medicine, McGill University; A. Iliescu, BS, Lady Davis Institute, Jewish General Hospital, Montreal; M. Baron, MD, Division of Rheumatology, Jewish General Hospital, and the Lady Davis Institute, Jewish General Hospital, and the Department of Medicine, McGill University; R. Steele, PhD, Lady Davis Institute, Jewish General Hospital, Montreal, and Mathematics and Statistics, McGill University.
J Rheumatol. 2016 Aug;43(8):1510-8. doi: 10.3899/jrheum.151440. Epub 2016 Jun 15.
To develop a weighted summary score for the Medsger Disease Severity Scale (DSS) and to compare its measurement properties with those of a summed DSS score and a physician's global assessment (PGA) of severity score in systemic sclerosis (SSc).
Data from 875 patients with SSc enrolled in a multisite observational research cohort were extracted from a central database. Item response theory was used to estimate weights for the DSS weighted score. Intraclass correlation coefficients (ICC) and convergent, discriminative, and predictive validity of the 3 summary measures in relation to patient-reported outcomes (PRO) and mortality were compared.
Mean PGA was 2.69 (SD 2.16, range 0-10), mean DSS summed score was 8.60 (SD 4.02, range 0-36), and mean DSS weighted score was 8.11 (SD 4.05, range 0-36). ICC were similar for all 3 measures [PGA 6.9%, 95% credible intervals (CrI) 2.1-16.2; DSS summed score 2.5%, 95% CrI 0.4-6.7; DSS weighted score 2.0%, 95% CrI 0.1-5.6]. Convergent and discriminative validity of the 3 measures for PRO were largely similar. In Cox proportional hazards models adjusting for age and sex, the 3 measures had similar predictive ability for mortality (adjusted R(2) 13.9% for PGA, 12.3% for DSS summed score, and 10.7% DSS weighted score).
The 3 summary scores appear valid and perform similarly. However, there were some concerns with the weights computed for individual DSS scales, with unexpected low weights attributed to lung, heart, and kidney, leading the PGA to be the preferred measure at this time. Further work refining the DSS could improve the measurement properties of the DSS summary scores.
制定梅兹格疾病严重程度量表(DSS)的加权总分,并将其测量属性与系统性硬化症(SSc)中DSS总分及医生整体严重程度评估(PGA)的测量属性进行比较。
从一个中央数据库中提取了参与多中心观察性研究队列的875例SSc患者的数据。采用项目反应理论估计DSS加权分数的权重。比较了这3种汇总测量方法与患者报告结局(PRO)和死亡率相关的组内相关系数(ICC)以及收敛效度、区分效度和预测效度。
PGA的平均值为2.69(标准差2.16,范围0 - 10),DSS总分的平均值为8.60(标准差4.02,范围0 - 36),DSS加权分数的平均值为8.11(标准差4.05,范围0 - 36)。这3种测量方法的ICC相似[PGA为6.9%,95%可信区间(CrI)2.1 - 16.2;DSS总分2.5%,95% CrI 0.4 - 6.7;DSS加权分数2.0%,95% CrI 0.1 - 5.6]。这3种测量方法对PRO的收敛效度和区分效度基本相似。在调整年龄和性别的Cox比例风险模型中,这3种测量方法对死亡率的预测能力相似(PGA调整后的R²为13.9%,DSS总分为12.3%,DSS加权分数为10.7%)。
这3种汇总分数似乎都是有效的,且表现相似。然而,对于为各个DSS量表计算的权重存在一些担忧,肺、心脏和肾脏的权重意外较低,导致目前PGA是首选测量方法。进一步完善DSS的工作可能会改善DSS汇总分数的测量属性。