表皮松解症临床研究评分工具（iscorEB）的信度和效度。
Reliability and validity of the instrument for scoring clinical outcomes of research for epidermolysis bullosa (iscorEB).
机构信息
Department of Dermatology, University of Colorado School of Medicine, Aurora, CO, U.S.A.
Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO, U.S.A.
出版信息
Br J Dermatol. 2018 May;178(5):1128-1134. doi: 10.1111/bjd.16350. Epub 2018 Apr 2.
BACKGROUND
Epidermolysis bullosa (EB) is a group of rare and currently incurable genetic blistering disorders. As more pathogenic-driven therapies are being developed, there is an important need for EB-specific validated outcomes measures designed for use in clinical trials.
OBJECTIVES
To test the reliability and construct validity of an instrument for scoring clinical outcomes of research for EB (iscorEB), a new combined clinician- and patient-reported outcomes tool.
METHODS
We conducted an observational study consisting of independent 1-day assessments (six assessors) at two academic hospitals. The assessments consisted of iscorEB clinician (iscorEB-c), Birmingham Epidermolysis Bullosa Severity (BEBS) and global severity assessment for physicians; and iscorEB patient (iscorEB-p), Quality of Life evaluation in Epidermolysis Bullosa and Children's Dermatology Life Quality Index for patients. Construct validity and intraclass correlation coefficients (ICCs) for interobserver, intraobserver and test-retest reliability were calculated.
RESULTS
Overall, 31 patients with a mean age of 19·5 years (1·8-45·2) were included. Disease severity was mild in 42% of cases, moderate in 29% and severe in 29%. The interobserver ICC was 0·96 for both the clinician-reported section of iscorEB-c and BEBS. The ICC for intraobserver reliability was 0·91 and 0·70 for the skin and mucosal domains of iscorEB-c, respectively. Cronbach's alpha for iscorEB-c was 0·89. The test-retest reliability of iscorEB-p was 0·97 and Cronbach's alpha was 0·84. The clinical score differentiated between subjects with mild, moderate and severe disease, and both clinical and patient subscores discriminated between recessive dystrophic EB and other EB subtypes.
CONCLUSIONS
iscorEB has robust reliability and construct validity, including strong ability to distinguish EB types and severities. Further studies are planned to test its responsiveness to change.
背景
大疱性表皮松解症（EB）是一组罕见且目前无法治愈的遗传性水疱性疾病。随着更多以致病机制为导向的治疗方法的发展，迫切需要设计专门用于临床试验的 EB 特异性验证结局测量工具。
目的
测试一种用于评估 EB 研究临床结局的新的综合临床医生和患者报告结局工具 iscorEB 的可靠性和结构效度。
方法
我们进行了一项观察性研究，包括在两所学术医院进行的独立 1 天评估（6 名评估者）。评估包括 iscorEB 临床医生（iscorEB-c）、伯明翰大疱性表皮松解症严重程度（BEBS）和医生的总体严重程度评估；以及 iscorEB 患者（iscorEB-p）、大疱性表皮松解症患者的生活质量评估和儿童皮肤病生活质量指数。计算了观察者间、观察者内和测试-重测信度的结构效度和组内相关系数（ICC）。
结果
共有 31 名年龄 19.5 岁（1.8-45.2）的患者纳入研究。42%的患者疾病严重程度为轻度，29%为中度，29%为重度。iscorEB-c 和 BEBS 的临床医生报告部分的观察者间 ICC 均为 0.96。iscorEB-c 的皮肤和黏膜域的观察者内信度 ICC 分别为 0.91 和 0.70。iscorEB-c 的克朗巴赫α系数为 0.89。iscorEB-p 的测试-重测信度为 0.97，克朗巴赫α系数为 0.84。临床评分可区分轻度、中度和重度疾病患者，临床和患者亚评分均可区分隐性营养不良性 EB 和其他 EB 亚型。
结论
iscorEB 具有可靠的可靠性和结构效度，包括区分 EB 类型和严重程度的强大能力。计划进一步研究以测试其对变化的反应能力。
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