Research Information System
Comprehensive medicine, vol.17, no.4, pp.301-308, 2025 (TRDizin)
Objective: : Localized scleroderma is a rare disease that can cause significant morbidity. Due to its rarity, data are limited, and no standardized treatment exists for patients resistant to first-line therapies. This study aims to evaluate clinical and treatment features based on a referral pediatric rheumatology center’s experience. Materials and Methods: The medical files of 36 included pediatric localized scleroderma patients were retrospectively reviewed, and demographic, clinical, laboratory, treatment, and outcome data were recorded. Results: Of the patients, 30 (83.3%) were female, the median age at diagnosis was 6.37 years (IQR: 4.04), and the median follow-up duration was 25.5 months (IQR: 56). The most common subtype was linear scleroderma (n=15, 41.67%), followed by circumscribed morphea (n=14, 38.89%). Among patients with linear scleroderma, 6 had craniofacial involvement. ANA positivity was observed in 38.9% of patients. Extracutaneous findings in the form of joint contractures were present in three patients. Systemic treatment was administered to 94% of patients, with methotrexate (MTX) used in 33 (91.67%) and corticosteroids in 26 (72.22%). MTX was effective in 58%, though some patients required additional or alternative therapies such as mycophenolate mofetil (n=14, 38.89%), intrave- nous immunoglobulin (n=9, 25.0%), or tocilizumab (n=2, 5.56%). Relapses occurred in 19%, and complete clinical response was achieved in 61%. Side effects were mostly mild and mainly related to MTX. Conclusion: Pediatric localized scleroderma shows diverse clinical presentations and often requires systemic treatment, primarily MTX. While most patients respond well, a subset needs additional therapies. Early diagnosis and tailored treatment are essential to improve outcomes and reduce morbidity.