Clinical Trial Leads to Approval for Tofacitinib to Treat Children With pcJIA

Published November 2021 | The Lancet

The Janus kinase (JAK) inhibitor tofacitinib is available today as an effective, affordable treatment for children with polyarticular course juvenile idiopathic arthritis (pcJIA) thanks in large part to a Phase 3 clinical trial co-coordinated by experts at Cincinnati Children’s.

Until now, treating JIA typically required administering intravenous or subcutaneous doses of synthetic and biological drugs. This international study was the first to evaluate a JAK inhibitor for this form of JIA, which affects five or more joints. Among the potentially exciting benefits: this disease modifying anti-rheumatic can be delivered orally.

The double-blind study included 225 children treated at 64 centers in 14 countries. At 44 weeks post-treatment, 53% of those receiving tofacitinib experienced reduced disease flares, compared to 29% of those receiving placebo.

Tofacitinib was approved in 2012 for adults with rheumatoid arthritis, but further study was needed to evaluate its effects in children. Based on data from this trial, the US FDA approved the drug for children in September 2020. European Commission approval occurred in August 2021. Full study results were published about three months later by The Lancet.

The study was organized by the Paediatric Rheumatology International Trials Organisation (PRINTO) and Pediatric Rheumatology Collaborative Study Group (PRCSG). The PRCSG, launched in 1973, was led for many years by Daniel Lovell, MD. Now, Hermine Brunner, MD, director, Division of Rheumatology, serves as its chair and scientific director. Brunner and Lovell were two of the four senior leaders on the study.

“Although there are already several advanced treatments available, oral tofacitinib will be an appealing new option. Injected and infused treatments can be quite burdensome to both children with pcJIA and their caretakers,” Brunner says.