EULAR 系统性硬化症疾病影响（ScleroID）问卷作为弥漫性系统性硬化症患者的患者报告结局测量工具的性能。
Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis.
机构信息
Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland.
Center of Experimental Rheumatology, University of Zurich, Zurich, Switzerland.
出版信息
RMD Open. 2024 Nov 27;10(4):e004653. doi: 10.1136/rmdopen-2024-004653.
OBJECTIVE
Systemic sclerosis Impact of Disease (ScleroID) is the first comprehensive patient-reported outcome measure (PROM) specifically developed for systemic sclerosis (SSc). We investigated the performance of ScleroID in patients with diffuse cutaneous SSc (dcSSc), as a prerequisite for its use in randomised controlled trials (RCTs) testing potentially disease-modifying drugs.
METHODS
All patients with dcSSc from the large, multicentric, ScleroID cohort were included. SSc-Health Assessment Questionnaire (HAQ), EuroQol-5 Dimensions and 36-item Short Form Health Survey (SF-36) were used as comparators. The study includes a longitudinal arm with a reliability visit at 7±3 days and a 12 months follow-up visit. The performance of ScleroID in dcSSc was assessed according to the Outcome Measures in Rheumatology filter.
RESULTS
In total, 152 dcSSc patients were analysed (29% male, median age 54 years). ScleroID reflected well the disease impact of dcSSc, showing a good construct validity with high Spearman's correlation coefficients with comparators (SSc-HAQ, 0.79, 95% CI (0.69, 0.86); HAQ-Disability Index, 0.72 95% CI (0.60, 0.80); SF-36 physical score, -0.69 95% CI (-0.77, -0.60)). The internal consistency was strong (Cronbach's alpha 0.87, split-half reliability coefficient 0.88).In the longitudinal arm, 44 patients had a reliability visit and 113 had a follow-up visit, of whom 19/113 (17%) reported a significant change (11 improved, 8 worsened). ScleroID showed a good consistency and discriminative ability with excellent test-retest reliability (intraclass correlation coefficient 0.89, 95% CI (0.84, 0.92)) and moderate sensitivity to change (standardised response mean -0.63 in the improved subgroup and 0.48 in the worsened subgroup), but superior to the comparators.
CONCLUSION
The European Alliance of Associations for Rheumatology (EULAR) ScleroID performs well for patients with dcSSc. This supports its inclusion and regular assessment as PROM in RCTs.
目的
系统性硬化症影响疾病（ScleroID）是第一个专门为系统性硬化症（SSc）开发的综合患者报告结局（PROM）测量工具。我们研究了 ScleroID 在弥漫性皮肤型 SSc（dcSSc）患者中的表现，这是其在测试潜在疾病修饰药物的随机对照试验（RCT）中使用的前提。
方法
所有来自大型多中心 ScleroID 队列的 dcSSc 患者均被纳入研究。将 SSc-健康评估问卷（HAQ）、EuroQol-5 维度和 36 项简明健康调查量表（SF-36）作为对照。该研究包括一个纵向部分，其中包括在 7±3 天进行可靠性访视和 12 个月随访访视。根据风湿病学结局测量过滤器评估 ScleroID 在 dcSSc 中的性能。
结果
共分析了 152 例 dcSSc 患者（29%为男性，中位年龄 54 岁）。ScleroID 很好地反映了 dcSSc 的疾病影响，与对照具有较高的斯皮尔曼相关系数，具有较好的结构效度（SSc-HAQ，0.79，95%CI（0.69，0.86）；HAQ 残疾指数，0.72 95%CI（0.60，0.80）；SF-36 身体评分，-0.69 95%CI（-0.77，-0.60））。内部一致性很强（Cronbach's alpha 0.87，半分可靠性系数 0.88）。在纵向部分，44 例患者进行了可靠性访视，113 例患者进行了随访，其中 19/113（17%）报告了显著变化（11 例改善，8 例恶化）。ScleroID 具有良好的一致性和判别能力，具有极好的测试-重测可靠性（组内相关系数 0.89，95%CI（0.84，0.92））和对变化的适度敏感性（改善亚组的标准化反应均值为-0.63，恶化亚组为 0.48），但优于对照。
结论
欧洲风湿病协会联盟（EULAR）的 ScleroID 在 dcSSc 患者中表现良好。这支持将其纳入并作为 RCT 中的 PROM 进行常规评估。
Zotero 插件