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Methotrexate in Linear Scleroderma: Long-Term Efficacy in Fifty Children From a Single Pediatric Rheumatology Center
Gloria Fadanelli1, Anna Agazzi2, Fabio Vittadello2
1Santa Chiara Hospital, Trento, Italy.
Insights
Methotrexate (MTX) treatment leads to long-lasting remission in most children with linear scleroderma (SSc). Early diagnosis and consistent monitoring are key for managing disease flares and improving outcomes.
Area of Science:
- Pediatric Rheumatology
- Dermatology
- Immunology
Background:
- Linear scleroderma (SSc) is a chronic autoimmune condition affecting children.
- Methotrexate (MTX) is a common immunosuppressive agent used in pediatric rheumatology.
- Understanding the long-term outcomes of MTX treatment in pediatric linear SSc is crucial for optimizing patient care.
Purpose of the Study:
- To evaluate the disease course and long-term outcomes of children with linear SSc treated with methotrexate (MTX).
- To assess the efficacy of MTX in achieving remission and preventing disease progression in pediatric linear SSc.
Main Methods:
- Retrospective, cross-sectional study including children with linear SSc treated with MTX for over one year and followed for at least two years.
- Disease course analyzed by relapse rates and treatment changes; relapse-free survival assessed using Kaplan-Meier analysis.
- Disease activity and damage evaluated using the Localized Scleroderma Cutaneous Assessment Tool and thermography.
Main Results:
- Fifty patients were included, with a mean follow-up of 7.8 years and MTX treatment of 3.1 years.
- Complete remission rates increased with follow-up duration: 18.2% (2-5 years), 80.0% (10 years), and 87.5% (>10 years).
- Longer treatment duration correlated significantly with fewer relapses and less tissue damage.
Conclusions:
- Methotrexate (MTX) treatment is effective in achieving complete and sustained remission in the majority of pediatric linear scleroderma cases.
- While moderate aesthetic and functional sequelae can occur, likely due to early damage, MTX appears to stabilize existing damage.
- Early diagnosis, prompt MTX initiation, and continuous monitoring are vital for improving long-term outcomes in children with linear SSc.
Objective:
To study disease course and long-term outcome in children with linear scleroderma (SSc) treated with methotrexate (MTX) since diagnosis.
Methods:
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.
Results:
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.
Conclusion:
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.
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