Efficacy and safety of tocilizumab in a real-life observational cohort of patients with polyarticular juvenile idiopathic arthritis

Abstract

Objectives To evaluate the patterns of usage, efficacy and safety of tocilizumab in polyarticular JIA. Methods An observational study of 56 consecutive polyarticular JIA patients was conducted using patient charts and electronic JIA databases. Efficacy was assessed by tocilizumab survival, rates of low disease activity (LDA) and of inactive disease by 10-joint Juvenile Arthritis Disease Activity Score (JADAS-10), and of clinically inactive disease according to Wallace’s preliminary criteria. Efficacy and rate of adverse events (AEs) were evaluated during a 24-month period after tocilizumab commencement. Results Tocilizumab was started on average as third-line biological agent (median, range first- to fourth-line) at a median disease duration of 5.2 years (interquartile range 3.0–7.7). Survival rates were 82% at 12 months and 64% at 24 months. The reasons for discontinuation were inadequate treatment effect in 50%, AE plus inadequate treatment effect in 37.5% and AE alone in 12.5%. LDA (JADAS-10 ⩽3.9) was reached in 58% at 12 months and in 84% at 24 months, inactive disease (JADAS-10 ⩽0.7) in 19% and 44%, and clinically inactive disease in 28% and 46%, respectively. The rate of AEs was 200.9/100 patient years and of serious AEs 12.9/100 patient years. Conclusion Survival of tocilizumab was high and a large proportion of the treatment-resistant patients reached LDA at 12 months of treatment. The LDA rate continued to increase throughout 24 months. The rates of AEs and serious AEs were higher than in register studies but lower than in the originator study of tocilizumab.

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

“…Interestingly, the effectiveness of TCZ in both sJIA and pJIA has been exemplified in other reports using variable scores [8,10,11,13,26,27].…”

“…Some patients exhibit no or minimal response after about 3-6 months of use of DMARDs which moreover show some annoying side effects and toxicities. Such patients are candidates for starting biological therapy [8]. Many biological agents seem to be effective in achieving minimal disease activity and improvement of long-term prognosis [9].…”

The clinical and paraclinical efficacy of tocilizumab in juvenile idiopathic arthritis

“…The average disease duration was 5.2 years. Juvenile Arthritis Disease Activity Score-10 (JADAS-10) of 3.9 was reported in 58% of cases at 12 months and in 84% of cases at 24 months; inactive disease (JADAS-10 of 0.7) was found in 19% of cases at 12 months and 44% at 24 months; the clinically inactive disease was registered in 28% of cases at 12 months and in 46% at 24 months 25 .…”

Biologics in Juvenile Idiopathic Arthritis-Main Advantages and Major Challenges: A Narrative Review

“…Apart from controlling disease activity in sJIA patients, tocilizumab has been reported to have therapeutic benefits in disease complications including secondary amyloidosis. [47][48][49][50][51] Tocilizumab was approved for IV injections in the treatment of patients with sJIA aged two to 17 years. Recently, tocilizumab treatment was investigated in sJIA patients younger than two years in an open-label phase 1 trial.…”