Ruperto N, Brunner HI, Synoverska O, et al. - Findings of this pivotal trial demonstrate effectiveness of tofacitinib (an oral Janus kinase inhibitor) as a treatment option for patients with polyarticular course juvenile idiopathic arthritis (JIA). New oral therapies are especially relevant for children and adolescents, who might prefer to avoid injections.

• Patients with polyarticular course JIA, aged 2 years to younger than 18 years, were studied in this double-blind, withdrawal phase 3 trial to evaluate the efficacy as well as safety of tofacitinib vs placebo in these cases.

• A total of 225 patients were enrolled, of which 184 (82%) had polyarticular course JIA, 20 (9%) had psoriatic arthritis, and 21 (9%) had enthesitis-related arthritis.

• Concomitant methotrexate was received by 147 (65%) of 225 patients, and part 2 of the study involved assignment of 142 patients with polyarticular course JIA to receive tofacitinib (n=72) or placebo (n=70).

• Tofacitinib afforded significantly lower flare rate by week 44 (21 [29%] of 72 patients) vs placebo (37 [53%] of 70 patients; hazard ratio 0·46).

• Part 2 of the study revealed occurrence of adverse events in 77% and 74% of patients in the tofacitinib and placebo groups, respectively.

• Occurrence of serious adverse events was noted in 1% and 2%, respectively, and 48% patients had infections or infestations in the entire tofacitinib exposure period.

• No deaths occurred during this study.