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Abstract
Background Since Tocilizumab has been approved for systemic onset juvenile idiopathic arthritis (soJIA) for ages 2 to 18 in 2011, children treated with Tocilizumab have been monitored prospectively in the German Biologics Register (BiKeR).
Objectives To evaluate the efficacy and safety in soJIA patients treated with Tocilizumab.
Methods Baseline demographics, clinical characteristics and disease activity parameters have been documented. Efficacy was determined using the PedACR 30/50/70 response criteria and JADAS. Safety assessments were based on adverse events reports from the primary responsible physician.
Results 29 sJIA patients (55% male) have been identified. Treatment with tocilizumab started at a mean (±SD) age of 9.9±5.3 years (1.5-19 years) after a disease duration of 5.8±4.7 years. Less than 30% started early, during the first 2 years of the disease. Numerous pre-treatments consisted of 39 courses of several immunosuppressive drugs (methotrexate 23, cyclosporine A 9, others 7) and 29 courses of several biologics (etanercept 18, anakina 7, canakinumab 2, abatacept 1, adalimumab 1 in 17 patients. Tocilizumab was the first biologic in 12 patients.
A high proportion of patients showed a significant response to treatment. Upon Tocilizumab, the number of swollen joints decreased by 91%, active joints by 78%, the patient’s global by 54%, physician’s global by 54%, the CHAQ-DI by 55%, the ESR by 79% and the CRP by 96%. At last observation after a mean (median) of 16 (11) months, the pedACR30/50/70 score was reached by 66%/66%/45% of patients.
The percentage of patients who met the criteria of 30%/50%/70% improvement at last documentation when Tocilizumab was the first introduced biological agent was 55%/55%/46% compared to 77%/77%/70% when Tocilizumab was initiated after failing to another biologics.
23 adverse events have been observed, mostly non-serious, including 3 medically important infections (herpes zoster, influenza, pneumonia), two cases with cytopenias, 2 patients with generalized urticaria. There were 3 serious adverse events (2 MAS, 1 herpes zoster), all resolved completely.
Conclusions Tocilizumab was efficacious in systemic JIA as a first and also as a second introduced biological agent. No new safety signals were noted. Infections, cytopenias and macrophage activation syndrome have been observed.
Acknowledgements This study would not have been possible without the collaboration of numerous German and Austrian pediatric rheumatologists, patients and their parents. The German Registry is supported by Abbott, Pfizer and Roche, Germany.
Disclosure of Interest G. Horneff Grant/research support from: Pfizer, Abbott, Roche, N. Onken: None Declared, A. Hospach: None Declared, G. Ganser: None Declared, H. Huppertz Consultant for: for Abbott, Pfizer, Roche, Swedish Orphan, Speakers bureau: Pfizer