Clinical efficacy and safety of sirolimus in childhood-onset systemic lupus erythematosus in real world

Objective: To investigate the effectiveness and safety of sirolimusin childhood onset systemic lupus erythematosus (cSLE) in a real world. Methods: : This is a retrospective real world clinical study. All cSLE patients treated with sirolimus in Children's Hospital of Hebei Province China were analyzed. They were treated with sirolimus and followed up regularly. The SLEDAI-2K score, levels of anti-double-stranded DNA (anti-dsDNA) antibody, complement components C3 and C4, and corticosteroid reduction were recorded at baseline and at 6, 12, and 18 months. Adverse events were also collected. Results: : 32 patients were enrolled in the study. SLEDAI-2K were improved on all time-points (p<0.05). Complement levels increased and the levels of anti-dsDNA antibody decreased during treatment. The mean dose of prednisone tapered and achieved significant reduction after 12 months therapy (15.4 ± 5.8 mg/day to 4.8 ± 2.1 mg/day; p<0.05). Sirolimus was well tolerated and only five patients (15.6%) experienced adverse events, all of which were classified as infections (two bacterial infection and three viral infections). No deaths, severe infusion reactions, or hypersensitivity reactions were found. Conclusions: : Sirolimus decreased disease activity and tapered daily glucocorticoid dose with a good safety profile.