Juvenile localized scleroderma: a large retrospective cohort study from a tertiary care center

Abstract

Abstract Background Juvenile localized scleroderma is a rare pediatric inflammatory disease that primarily affects the skin and subcutaneous tissue but also has the potential to impact deeper tissues and can be associated with extracutaneous manifestations, leading to substantial impairment and disability. Management approaches vary, but in recent years, expert groups have attempted to streamline the approach to care with consensus treatment plans. Methods This retrospective cohort study included pediatric juvenile localized scleroderma patients with ≥ 3 years of follow-up identified within a 21-year period (1999–2020) at a single tertiary care pediatric hospital in the USA. Data on demographics, disease characteristics, and treatment trends were analyzed, with a focus on systemic versus topical therapy and treatment trends before and after the publication of the Childhood Arthritis and Rheumatology Research Alliance juvenile localized scleroderma consensus treatment plan in 2012. Results A total of 101 juvenile localized scleroderma patients fulfilled our inclusion criteria. Sixty-three patients were treated with systemic therapy, and 38 were treated with topical therapy. Patients on systemic therapy were more commonly treated in a combined rheumatology-dermatology program (67%) or rheumatology clinic (30%), whereas those on topical therapy were primarily treated in a dermatology clinic (71%). Starting in 2013, a significantly greater percentage of all patients were treated in the combined program (47% vs. 20%, p = 0.008), and a significantly greater percentage of patients received systemic therapy (78% vs. 55%, p < 0.05). Conclusion This juvenile localized scleroderma cohort is one of the largest reported from a single center and demonstrated an increase in the use of systemic therapy following the publication of the Childhood Arthritis and Rheumatology Research Alliance juvenile localized scleroderma consensus treatment plan in 2012. Further studies on long-term treatment outcomes and therapeutic approaches utilized when first-line treatment failures occur are warranted.

Keywords

• Scleroderma
• Treatment
• Rheumatology
• Dermatology
• Juvenile localized scleroderma
• Consensus treatment plans