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Background Juvenile idiopathic arthritis (JIA) is the most prevalent pediatric rheumatic disorder. Progress in modalities of therapy improves the disease outcome. We aimed to determine the efficacy and safety of tocilizumab (TCZ) in the management of systemic (sJIA) and polyarticular (pJIA) in children who are resistant to conventional as well as other biological therapies. In this retrospective study, we selected JIA patients according to the International League of Association for Rheumatology (ILAR) criteria and were treated with TCZ. Response to TCZ was assessed using Juvenile Arthritis Disease Activity Score-10 (JADAS-10) and also outcomes were assessed according to the American College of Rheumatology (ACR) remission criteria. Safety of the drug was assessed by documenting possibly related adverse effects (AE). Statistical analysis using SPSS version 25 with statistical significance is considered if  p  ≤ 0.05. We included 16 JIA patients aged ≤ 18 years but 2 of them were excluded as they developed severe reaction during the TCZ 1st dose, so finally, 14 patients were included. Results The median age of our patients was 12 years. Of these 14 patients, 9 (64.3%) had sJIA and 5 (35.7%) had pJIA. TCZ use led to significant improvement in the JADAS-10 from mean 22.4 (± 7.9) when it was initially assessed and then 3, 6, 12, and 24 months after TCZ initiation with means 5.7 (± 3.9), 4.4 (± 3.7), 3.5 (± 3.1), and 2.7 (± 2.2), ( P  = 0.001, 0.001, 0.005, 0.012), respectively. Five patients exhibited TCZ possibly expected side effects. Neutropenia and infusion-related reactions were the most frequent AE. Conclusions Tocilizumab seems to be generally effective and safe drug in the management of sJIA and pJIA especially in cases refractory to conventional as well as other biologic agents.

Tocilizumab in management of refractory juvenile idiopathic arthritis, 10 years of experience at tertiary university hospital