Klotsche Jens, Torok Kathryn S, Kasapcopur Ozgur, Adrovic Amra, Terreri Maria Teresa, Sakamoto Ana Paula, Katsicas Maria, Sztajnbok Flavio, Marrani Edoardo, Sifuentes-Giraldo Alberto, Stanevicha Valda, Anton Jordi, Feldmann Brian, Kostik Mikhail, Nemcova Dana, Santos Maria Jose, Appenzeller Simone, Avcin Tadej, Battagliotti Cristina, Berntson Lillemor, Bica Blanca, Brunner Jürgen, Eleftheriou Despina, Harel Liora, Horneff Gerd, Kallinich Tilmann, Minden Kirsten, Nielsen Susan, Patwardhan Anjali, Helmus Nicola, Foeldvari Ivan
German Rheumatism Research Center, A Leibniz Institute, Berlin, Germany.
University of Pittsburgh, Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
J Scleroderma Relat Disord. 2023 Oct;8(3):183-191. doi: 10.1177/23971983231164700. Epub 2023 Apr 10.
Juvenile systemic sclerosis is a rare childhood disease. Three disease activity indices have been published for adult patients with systemic sclerosis: the European Scleroderma Study Group Index, a modified version of the European Scleroderma Study Group Index and the revised European Scleroderma Trials and Research index. The objective of this study was to determine the feasibility and performance of the three disease activity indices in a prospectively followed cohort of patients with juvenile systemic sclerosis.
The analysis cohort was selected from the prospective international inception cohort enrolling juvenile systemic sclerosis patients. The correlation of the disease activity indices with the physicians' and the patients' global assessment of disease activity was determined. The disease activity indices were compared between patients with active and inactive disease. Sensitivity to change between 6- and 12-month follow-up was investigated by mixed models.
Eighty percent of the 70 patients had a diffuse cutaneous subtype. The revised European Scleroderma Trials and Research index was highly correlated with the physician-reported global disease activity/parents-reported global disease activity (r = 0.74/0.64), followed by the European Scleroderma Study Group activity index (r = 0.61/0.55) and the modified version of the European Scleroderma Study Group activity index (r = 0.51/0.43). The disease activity indices significantly differed between active and inactive patients. The disease activity indices showed sensitivity to change between 6- and 12-month follow-up among patients who improved or worsened according to the physician-reported global disease activity and the parents-reported global disease activity.
Overall, no disease activity score is superior to the other, and all three scores have limitations in the application in juvenile systemic sclerosis patients. Furthermore, research on the concept of disease activity and suitable scores to measure disease activity in patients with juvenile systemic sclerosis is necessary in future.
青少年系统性硬化症是一种罕见的儿童疾病。针对成年系统性硬化症患者已发布了三种疾病活动指数：欧洲硬皮病研究组指数、欧洲硬皮病研究组指数的修订版以及修订后的欧洲硬皮病试验与研究指数。本研究的目的是确定这三种疾病活动指数在一个前瞻性随访的青少年系统性硬化症患者队列中的可行性和表现。
分析队列选自招募青少年系统性硬化症患者的前瞻性国际初始队列。确定疾病活动指数与医生和患者对疾病活动的整体评估之间的相关性。比较活动期和非活动期患者的疾病活动指数。通过混合模型研究6个月和12个月随访期间对变化的敏感性。
70名患者中有80%为弥漫性皮肤亚型。修订后的欧洲硬皮病试验与研究指数与医生报告的整体疾病活动/父母报告的整体疾病活动高度相关（r = 0.74/0.64），其次是欧洲硬皮病研究组活动指数（r = 0.61/0.55）和欧洲硬皮病研究组活动指数的修订版（r = 0.51/0.43）。活动期和非活动期患者的疾病活动指数有显著差异。根据医生报告的整体疾病活动和父母报告的整体疾病活动，疾病活动指数在改善或恶化的患者6个月和12个月随访期间显示出对变化的敏感性。
总体而言，但没有一种疾病活动评分优于其他评分，并且所有三种评分在青少年系统性硬化症患者中的应用都有局限性。此外，未来有必要对青少年系统性硬化症患者疾病活动的概念和测量疾病活动的合适评分进行研究。
