Clinical characteristics, autoantibody profiles, and therapeutic outcomes of juvenile systemic sclerosis: an 11-year single-center experience from China.

OBJECTIVE

Juvenile systemic sclerosis (jSSc) is a rare but serious rheumatic disease in children that significantly impacts growth and development. This study aimed to summarize the clinical characteristics and disease patterns of jSSc and to evaluate the treatment response over an 11-year period at our center.

METHODS

Twenty-one pediatric jSSc patients treated at our center from January 2015 to December 2025 were retrospectively analyzed. Classification was based on the 2013 American College of Rheumatology (ACR)/European Alliance of Associations for Rheumatology (EULAR) systemic sclerosis classification criteria. Overall disease severity and multiorgan activity were assessed using the Juvenile Systemic Sclerosis Severity Score (J4s; range 0-40), while skin involvement was specifically evaluated using the modified Rodnan skin score (mRSS; range 0-51). The median follow-up duration was 2.3 years (range 0.5-7.2 years). Paired t-tests and Wilcoxon signed-rank tests were used to compare pre- and posttreatment parameters, with p < 0.05 considered statistically significant.

RESULTS

Twenty-one patients were enrolled. Skin sclerosis was the most common symptom (100.0%). Antinuclear antibody (ANA) was positive in 16 cases (76.2%), with anti-single-stranded DNA (anti-ssDNA) antibody being the most common specific antibody (23.8%), followed by anti-PM-Scl antibody (19.0%). Glucocorticoids and methotrexate were commonly used as initial therapies. After a median treatment duration of 24 months, overall disease severity (J4s) significantly decreased (5.94 ± 1.66 vs 2.67 ± 2.22, p = 0.003), and skin involvement (mRSS) also improved significantly (10.0 ± 9.21 vs 7.42 ± 8.26, p = 0.008). Fourteen patients received biologic agents, with 11 using tocilizumab. During the follow-up, no deaths or serious adverse events occurred.

CONCLUSIONS

jSSc is a rare and severe multiorgan disease with unique autoantibody profiles in Chinese children. The J4s and mRSS serve as objective assessment tools for evaluating overall disease severity and skin involvement, respectively, and are effective in monitoring treatment response. Early diagnosis and individualized therapy, including the adjunctive use of tocilizumab, may improve clinical outcomes.