Becker Mike O, Dobrota Rucsandra, Garaiman Alexandru, Debelak Rudolf, Fligelstone Kim, Tyrrell Kennedy Ann, Roennow Annelise, Allanore Yannick, Carreira Patricia E, Czirják László, Denton Christopher P, Hesselstrand Roger, Sandqvist Gunnel, Kowal-Bielecka Otylia, Bruni Cosimo, Matucci-Cerinic Marco, Mihai Carina, Gheorghiu Ana Maria, Mueller-Ladner Ulf, Sexton Joseph, Kvien Tore K, Heiberg Turid, Distler Oliver
Department of Rheumatology, University Hospital of Zurich, Zurich, Switzerland.
Department of Psychology, Psychological Methods, Evaluation and Statistics, University of Zurich, Zurich, Switzerland.
Ann Rheum Dis. 2022 Apr;81(4):507-515. doi: 10.1136/annrheumdis-2021-220702. Epub 2021 Nov 25.
Patient-reported outcome measures (PROMs) are important for clinical practice and research. Given the high unmet need, our aim was to develop a comprehensive PROM for systemic sclerosis (SSc), jointly with patient experts.
This European Alliance of Associations for Rheumatology (EULAR)-endorsed project involved 11 European SSc centres. Relevant health dimensions were chosen and prioritised by patients. The resulting Systemic Sclerosis Impact of Disease (ScleroID) questionnaire was subsequently weighted and validated by Outcome Measures in Rheumatology criteria in an observational cohort study, cross-sectionally and longitudinally. As comparators, SSc-Health Assessment Questionnaire (HAQ), EuroQol Five Dimensional (EQ-5D), Short Form-36 (SF-36) were included.
Initially, 17 health dimensions were selected and prioritised. The top 10 health dimensions were selected for the ScleroID questionnaire. Importantly, Raynaud's phenomenon, impaired hand function, pain and fatigue had the highest patient-reported disease impact. The validation cohort study included 472 patients with a baseline visit, from which 109 had a test-retest reliability visit and 113 had a follow-up visit (85% female, 38% diffuse SSc, mean age 58 years, mean disease duration 9 years). The total ScleroID score showed strong Pearson correlation coefficients with comparators (SSc-HAQ, 0.73; Patient's global assessment, Visual Analogue Scale 0.77; HAQ-Disability Index, 0.62; SF-36 physical score, -0.62; each p<0.001). The internal consistency was strong: Cronbach's alpha was 0.87, similar to SSc-HAQ (0.88) and higher than EQ-5D (0.77). The ScleroID had excellent reliability and good sensitivity to change, superior to all comparators (intraclass correlation coefficient 0.84; standardised response mean 0.57).
We have developed and validated the EULAR ScleroID, which is a novel, brief, disease-specific, patient-derived, disease impact PROM, suitable for research and clinical use in SSc.
患者报告的结局指标（PROMs）对临床实践和研究很重要。鉴于未满足的需求较高，我们的目标是与患者专家共同开发一种用于系统性硬化症（SSc）的综合PROM。
这个得到欧洲风湿病协会联盟（EULAR）认可的项目涉及11个欧洲SSc中心。由患者选择相关健康维度并确定其优先级。随后，在一项观察性队列研究中，按照风湿病结局指标标准对生成的系统性硬化症疾病影响（ScleroID）问卷进行加权和验证，包括横断面和纵向研究。作为对照，纳入了SSc健康评估问卷（HAQ）、欧洲五维健康量表（EQ-5D）、简短健康调查问卷（SF-36）。
最初，选择了17个健康维度并确定其优先级。为ScleroID问卷选择了前10个健康维度。重要的是，雷诺现象、手部功能受损、疼痛和疲劳在患者报告的疾病影响方面最为突出。验证队列研究包括472例进行了基线访视的患者，其中109例进行了重测信度访视，113例进行了随访（85%为女性，38%为弥漫性SSc，平均年龄58岁，平均病程9年）。ScleroID总分与对照指标显示出很强的皮尔逊相关系数（与SSc-HAQ相关系数为0.73；患者整体评估，视觉模拟量表为0.77；HAQ残疾指数为0.62；SF-36身体评分，-0.62；各p<0.001）。内部一致性很强：Cronbach's α为0.87，与SSc-HAQ（0.88）相似，高于EQ-5D（0.77）。ScleroID具有出色的信度和良好的变化敏感性，优于所有对照指标（组内相关系数0.84；标准化反应均值0.57）。
我们已经开发并验证了EULAR ScleroID，这是一种新颖、简短、针对疾病、源自患者的疾病影响PROM，适用于SSc的研究和临床应用。
