To evaluate the efficacy and safety of baricitinib in pediatric patients with active juvenile idiopathic arthritis-associated uveitis (JIA-U) or chronic anterior antinuclear antibody-positive uveitis, who had an inadequate response to methotrexate (MTX) or biologic disease-modifying antirheumatic drugs (bDMARDs).

JUVE-BRIGHT was an open-label, active-controlled phase-3 multicenter trial that used 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 Standardization of Uveitis Nomenclature (SUN) criteria as a two-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 prespecified 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 pediatric patients. The study primary endpoint was not met. In the baricitinib group, 36.8% of MTX-inadequate responder (MTX-IR) and bDMARD-IR patients and 20% of MTX-IR patients achieved a two-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 pediatric and adult patients.

Although the primary endpoint was not met, the data provide 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.