Effectiveness and safety of baricitinib for juvenile idiopathic arthritis associated uveitis or chronic anterior antinuclear antibody positive uveitis

Evaluate the efficacy and safety of baricitinib in paediatric patients with active JIA-U or chronic anterior ANA-positive uveitis, who had an inadequate response to MTX or bDMARDs. JUVE-BRIGHT was an open-label, active-controlled, Phase-3 multicentre trial which utilized a novel design, including 1:1 randomization to an active reference arm. The primary efficacy endpoint was the proportion of responders at Week 24 (W24), defined according to the SUN criteria as a 2-step decrease in the level of inflammation (anterior chamber cells) or decrease to zero through W24 in the most severely affected eye at baseline. Study success was based on a pre-specified Bayesian success rule: the study was deemed successful if there was >80% posterior probability that the baricitinib SUN criteria response rate at W24 was at least 57%. This study enrolled 30 paediatric patients. The study primary endpoint was not met. In the baricitinib group, 36.8% of MTX-IR and bDMARD-IR and 20% of MTX-IR patients achieved a 2-step decrease in SUN criteria at W24. Eight patients (33.3%) achieved a response at W24, resulting in 1.03% posterior probability of a response rate of >57%. Safety data were consistent with the established safety profile in other baricitinib indications in paediatric and adult patients. Although the primary endpoint was not met, the data provides important information on baricitinib for the treatment of children with JIA-U refractory to both MTX and bDMARDs. Baricitinib safety profile in this study was consistent with previous studies in children and adults with other diseases.