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Clinical Characteristics and Treatment Patterns in Pediatric Localized Scleroderma: A Referral-center Experience

Year: 2025
Type: article
Study design: Observational Study
Abstract: 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%), intravenous 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.
Language: English
Field: Medicine
Open Access status: diamond
OpenAlex ID: W4414998000
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