Arkachaisri Thaschawee, Pino Sally
Division of Rheumatology, Children's Hospital of Pittsburgh, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania 15213, USA.
J Rheumatol. 2008 Apr;35(4):650-7. Epub 2008 Mar 1.
To develop a disease outcome instrument measuring localized scleroderma (LS) severity and to determine its reliability.
Patients with LS were evaluated by 2 rheumatologists. The LS Severity Index (LoSSI) comprises the sum of 4 clinical skin scores measured at 14 cutaneous anatomic sites: extent of surface area (SA) affected, erythema score (ES), skin thickness (ST) score, and new lesion/extension (N/E). Physician and patient global assessments (GA) were recorded on a 100 mm visual analog scale.
Twenty-two patients with LS had 66 visits, during which 91 lesions were assessed. Individual skin scores (SA, ES, ST) and LoSSI showed substantial interrater reliability (kappaw 0.77-0.83; percentage agreement 93.41%-96.70%). Intrarater variability was calculated using 26 anatomic sites and 9 pair-visits. Each skin score demonstrated excellent reliability (kappaw 0.56-0.80, percentage agreement 90.39%-94.23%). Physician GA showed substantial interrater correlation [0.72, 95% confidence interval (CI) 0.57, 0.87]. There was only fair correlation between physician and patient GA (0.27, 95% CI 0.00, 0.64). The standardized response means for LoSSI and physician GA were large (1.86 and 2.55) for those who improved after therapy.
LS clinical trials are impeded by the lack of reliable and reproducible outcome measures. We have developed the LoSSI to correct this deficiency. Our pilot study demonstrates that the LoSSI is reliable and reproducible in measuring LS severity and therapeutic effects and can be easily implemented into the clinical examination of patients with LS. Both LoSSI and physician GA were sensitive to clinical changes in patients with LS. A formal study should be conducted to validate these preliminary findings.
开发一种测量局限性硬皮病（LS）严重程度的疾病结局工具，并确定其可靠性。
由2名风湿病学家对LS患者进行评估。LS严重程度指数（LoSSI）包括在14个皮肤解剖部位测量的4项临床皮肤评分的总和：受影响的表面积（SA）范围、红斑评分（ES）、皮肤厚度（ST）评分以及新病变/扩展（N/E）。医生和患者的整体评估（GA）采用100毫米视觉模拟量表记录。
22例LS患者共就诊66次，期间评估了91个皮损。各项皮肤评分（SA、ES、ST）和LoSSI显示出较高的评分者间信度（kappa值为0.77 - 0.83；百分比一致性为93.41% - 96.70%）。使用26个解剖部位和9次配对就诊计算评分者内变异性。每项皮肤评分均显示出良好的信度（kappa值为0.56 - 0.80，百分比一致性为90.39% - 94.23%）。医生的GA显示出较高的评分者间相关性[0.72，95%置信区间（CI）为0.57，0.87]。医生和患者的GA之间仅有中等程度的相关性（0.27，95%CI为0.00，0.64）。治疗后病情改善者的LoSSI和医生GA的标准化反应均值较大（分别为1.86和2.55）。
缺乏可靠且可重复的结局测量方法阻碍了LS的临床试验。我们开发了LoSSI以纠正这一缺陷。我们的初步研究表明，LoSSI在测量LS严重程度和治疗效果方面可靠且可重复，并且可以轻松应用于LS患者的临床检查。LoSSI和医生GA对LS患者的临床变化均敏感。应进行正式研究以验证这些初步发现。
