The efficacy and safety of tocilizumab in juvenile localised scleroderma patients: a retrospective cohort study in...

Localised scleroderma is a rare autoimmune connective tissue disorder characterized by excessive collagen deposition leading to skin fibrosis. While methotrexate and glucocorticosteroid remain the first-line treatments, their limitations underscore the need to explore targeted biologic therapies. Tocilizumab, an IL-6 receptor antagonist, has shown promise in some systemic autoimmune diseases, but its role in juvenile localised scleroderma remains unclear. This study retrospectively evaluates the efficacy and safety of tocilizumab in juvenile localised scleroderma patients.

This retrospective study analysed 12 juvenile localised scleroderma patients treated at Anhui Province Children's Hospital between January 2021 and December 2024. Patients were grouped based on early versus delayed initiation of tocilizumab. Clinical response was measured using mLoSSI and LoSDI scores. Laboratory markers (ALT, AST, cholesterol, triglycerides, CRP) and immune indicators (CD4, CD19, IgG) were assessed alongside treatment duration. Safety and glucocorticosteroid usage were also evaluated.

Five patients received tocilizumab from treatment onset, while seven were treated later. The tocilizumab initial use group had a significant lower relapse rate, compared to the delayed group. Longer treatment with tocilizumab had a protective effect on liver function and lipid metabolism, due to less abnormal ALT and triglyceride. No statistically significant relationship was observed between IgG deficiency and the duration of tocilizumab therapy; only one patient developed IgG deficiency, which resolved after a three-week interruption of treatment. Tocilizumab also reduced glucocorticosteroid reuse and duration of usage.

Tocilizumab appears to be a safe and effective adjunctive therapy for juvenile localised scleroderma, particularly when initiated early, as it reduces relapse risk and the need for prolonged glucocorticosteroid use. It remains a promising adjunct in refractory or severe cases requiring long-term management.