Baricitinib showed limited efficacy in pediatric JIA-uveitis patients refractory to methotrexate or biologics at week 24.

OBJECTIVE: 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). METHODS: 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%. RESULTS: 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. CONCLUSION: 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.