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

Objectives: 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.

Methods: 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%.

Results: 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.

Conclusion: 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.