Department of Pediatrics, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Hackensack Meridian School of Medicine, Hackensack, NJ.
Department of Pediatrics, The Ohio State University and Nationwide Children's Hospital, Columbus, OH.
Rheumatology (Oxford). 2021 Dec 1;60(12):5724-5733. doi: 10.1093/rheumatology/keab238.
The aim of this study was to evaluate factors associated with extracutaneous involvement (ECI) in juvenile localized scleroderma (jLS).
A prospective, multicentre, 6-month observational study was performed. The data collected included disease features, global assessments, and subject symptoms. Bivariate and linear multilevel regression analyses were performed.
A total of 86 jLS subjects (80% female, 80% Caucasian), median age of disease onset 7.7 years, were evaluated. Most had linear scleroderma or mixed morphea. Of the 86 subjects, 49 (57%) had 125 extracutaneous problems {median 2 [interquartile range (IQR) 1, 3] per subject} from nine organ systems. Most of these subjects had multiple musculoskeletal problems. ECI was associated with more extensive cutaneous involvement, higher number of symptoms, family history of autoimmunity, and ANA and RF positivity. Subjects with ECI had higher scores for physician global assessment of damage (PGA-D), and parental global assessment of disease impact, but not baseline physician global assessment of disease activity (PGA-A). Although subjects with ECI received more MTX and glucocorticoid treatment, they had a slower reduction in PGA-A scores and symptoms over time, suggesting a poorer response to treatment. In logistic regression modelling, female sex had the largest effect on parental impact scores.
ECI occurred in the majority of subjects with jLS, and was associated with more medication use, longer treatment duration, higher PGA-D scores, and higher parental assessment of disease impact. Our findings suggest that jLS subjects with ECI have greater overall disease burden, both cutaneous and extracutaneous, and poorer response to treatment. More study of the treatment needs of this population is warranted.
本研究旨在评估与儿童局限性硬皮病（jLS）相关的皮肤外受累（ECI）的因素。
进行了一项前瞻性、多中心、6 个月的观察性研究。收集的数据包括疾病特征、整体评估和患者症状。进行了双变量和线性多级回归分析。
共评估了 86 名 jLS 患者（80%为女性，80%为白种人），发病年龄中位数为 7.7 岁。大多数患者为线性硬皮病或混合硬肿病。86 名患者中有 49 名（57%）有 125 个来自九个器官系统的皮肤外问题{中位数为 2 [四分位距（IQR）1，3]个/患者}。这些患者大多数有多发性肌肉骨骼问题。ECI 与更广泛的皮肤受累、更多的症状、自身免疫性疾病家族史以及抗核抗体和类风湿因子阳性相关。有 ECI 的患者的医生整体损伤评估（PGA-D）和父母对疾病影响的整体评估得分更高，但基线医生对疾病活动的整体评估（PGA-A）没有。尽管有 ECI 的患者接受了更多的 MTX 和糖皮质激素治疗，但他们的 PGA-A 评分和症状随时间的推移减少得更慢，这表明他们对治疗的反应较差。在逻辑回归模型中，女性性别对父母影响评分的影响最大。
jLS 患者中大多数有 ECI，与更多的药物使用、更长的治疗时间、更高的 PGA-D 评分以及父母对疾病影响的更高评估相关。我们的研究结果表明，有 ECI 的 jLS 患者具有更大的总体疾病负担，既有皮肤受累也有皮肤外受累，且对治疗的反应较差。需要进一步研究这一人群的治疗需求。
