Santa Chiara Hospital, Trento, Italy.
University of Padova, Padova, Italy.
Arthritis Care Res (Hoboken). 2021 Sep;73(9):1259-1263. doi: 10.1002/acr.24260. Epub 2021 Aug 4.
To study disease course and long-term outcome in children with linear scleroderma (SSc) treated with methotrexate (MTX) since diagnosis.
The present study was retrospective and cross-sectional and included consecutive children with linear SSc who were treated with MTX for >1 year and were followed up for at least 2 years. Disease course was analyzed by the number of relapses and treatment changes. Relapse-free survival was examined by Kaplan-Meier analysis, comparing patients with linear SSc and those with other juvenile localized scleroderma (JLS) disease subtypes. Disease activity and damage were assessed by the Localized Scleroderma Cutaneous Assessment Tool and thermography.
Fifty patients with a mean follow-up duration of 7.8 years and a mean MTX treatment duration of 3.1 years were included. Sixteen percent of patients did not respond to the first course of MTX, and 16% had at least 1 flare. Complete remission was observed in 18.2% of patients who were followed up for 2-5 years, in 80.0% of patients followed up for 10 years, and in 87.5% of patients followed up for >10 years. No significant difference in relapse-free survival between patients with linear SSc and in 17 patients with other JLS disease subtypes was observed. Tissue damage was mild in 42% of patients, moderate in 32%, and severe in 26%. The correlations between severity of tissue damage and linear SSc subtype, disease duration, relapses, and remission were not significant. The relationships between treatment duration and disease relapses (P < 0.05) and severity of tissue damage (P < 0.005) were significant.
Most patients with linear SSc who are treated with MTX achieve complete and long-lasting remission. Overall aesthetic and functional sequelae are moderate, most likely because tissue damage is established early and treatment likely stabilizes the damage. Early diagnosis and MTX treatment, as well as long-term monitoring, are crucial to improve outcome and promptly identify flares.
研究接受甲氨蝶呤（MTX）治疗的儿童线状硬皮病（SSc）的疾病进程和长期预后。
本研究为回顾性和横断面研究，纳入了接受 MTX 治疗>1 年且随访时间至少 2 年的连续儿童线状 SSc 患者。通过复发次数和治疗改变来分析疾病进程。通过 Kaplan-Meier 分析比较线性 SSc 患者和其他青少年局限性硬皮病（JLS）疾病亚型患者的无复发生存率。通过局限性硬皮病皮肤评估工具和热成像评估疾病活动度和损伤。
纳入 50 例患者，平均随访时间为 7.8 年，MTX 治疗时间平均为 3.1 年。16%的患者对第一疗程 MTX 无反应，16%的患者至少有 1 次发作。在随访 2-5 年的患者中，18.2%达到完全缓解，在随访 10 年的患者中，80.0%达到完全缓解，在随访>10 年的患者中，87.5%达到完全缓解。线性 SSc 患者与其他 17 例 JLS 疾病亚型患者之间无复发生存率差异无统计学意义。42%的患者组织损伤较轻，32%的患者组织损伤中度，26%的患者组织损伤严重。组织损伤严重程度与线性 SSc 亚型、疾病持续时间、复发和缓解之间无显著相关性。治疗持续时间与疾病复发（P<0.05）和组织损伤严重程度（P<0.005）之间存在显著关系。
大多数接受 MTX 治疗的儿童线状 SSc 患者达到完全和持久缓解。总体美观和功能后遗症为中度，这很可能是因为组织损伤较早发生，治疗可能稳定了损伤。早期诊断和 MTX 治疗以及长期监测对于改善预后和及时识别发作至关重要。
