Background/Purpose: Linear Scleroderma (LS) is the most common subtype of localized Scleroderma in children and the one most frequently associated with tissue damage and functional disability. During the last decade, methotrexate (MTX) has been used as first choice agent for LS but, to date, available data on long-term outcome are partial and incomplete. Aim of this study was to assess the disease recurrence and the long-term outcome of children with LS treated with MTX since diagnosis.

Methods: We conducted a retrospective and cross-sectional study including children with LS followed at our Paediatric Rheumatology Centre between 2000 and 2016. Patients treated with MTX for at least one year, then followed for at least 2 years were included. Disease course was evaluated by retrospective analysis of clinical features, treatment and number of flares. Outcome at the last visit was assessed by evaluation of disease activity and severity of tissue damage and/or functional impairment by Localized Scleroderma Cutaneous Assessment Tool (LoSCAT) and thermography. Clinical remission (CR) was defined in presence of inactive disease off treatment for more than two years; clinical remission on medication (CRM) in presence of inactive disease while on treatment.

Results: 50 patients, 24 with LS of the trunk/limbs and 26 with LS of the face, entered the study. MTX treatment duration was 3.1 years (median 2.9, range 1.8-8.5 years). Only 16% patients did not respond to the first therapy with MTX, 16% had at least one flare of the disease, on average 30 months after starting MTX and 10% presented recurrent relapses. The mean follow-up was 7.8 years (median 6.8, range 2.1-16.3). Within 5 year follow-up, 81.8% of patients achieved CRM and by 10 year follow-up, 80.0% obtained CR. In the group of 16 patients with >10 years, CR was reported in 87.5%. At the last evaluation, tissue damage was very mild in 42% of patients, moderate in 32% and severe in 26%. Various degree of functional limitation was reported in 41.7% of patients with LS of the limbs. Severity of tissue damage was not related to the part of the body involved or to the disease duration, while functional disability was more frequent in LS of the limbs (p=0.039). LS body site and severity of tissue damage were not associated with disease recurrence or remission, while the length of therapy was related to number of disease relapses (p=0.040) and severity of tissue damage (p=0.003).

Conclusion: Most patients with LS treated with MTX achieve complete remission without flares and only a minority present repeated relapses or active disease after more than 10 years. The long-term monitoring of the patients, even after stopping MTX treatment is crucial to promptly identify possible flares and treat them properly. Overall aesthetic and functional sequelae are moderate and develop early in the disease course as their severity is not related to the disease duration.

« Back to 2018 ACR/ARHP Annual Meeting

ACR Meeting Abstracts - https://acrabstracts.org/abstract/long-term-outcome-of-50-patients-with-linear-scleroderma-treated-with-methotrexate/