Tofacitinib for the Treatment of Juvenile Idiopathic Arthritis: Patient-Reported Outcomes in a Phase 3, Randomized, Double-Blind, Placebo-Controlled Withdrawal Trial

Objective: Juvenile idiopathic arthritis (JIA) is associated with impaired overall health-related quality of life (HRQoL). We evaluated the impact of tofacitinib on patient-reported outcomes (PROs) in patients with JIA. Methods: This was a post hoc analysis of a phase 3, randomized, double-blind, placebo-controlled withdrawal trial (NCT02592434) in patients with JIA. In the open-label phase (part 1; weeks 0–18), patients received body weight–based doses of tofacitinib. During the double-blind phase (part 2; weeks 18–44), responders (per JIA-American College of Rheumatology 30 response criteria) were randomized 1:1 to continue tofacitinib or switch to placebo for up to 26 weeks. Assessed PROs included the validated parent and/or legal guardian versions of the Childhood Health Assessment Questionnaire for evaluation of disability, arthritis pain, overall well-being, and the Child Health Questionnaire (CHQ). Results: Overall, 225 patients were enrolled and received open-label tofacitinib in part 1, and 173 patients were randomized in part 2. During part 1, least-squares (LS) mean (SE) disability, arthritis pain, and overall well-being scores numerically improved from mean 1.04 (SE 0.05), mean 5.53 (SE 0.20), and mean 5.07 (SE 0.20) at baseline to mean 0.57 (SE 0.05), mean 2.46 (SE 0.18), and mean 2.47 (SE 0.18) at week 18, respectively. LS mean (SE) CHQ physical summary and psychological summary scores numerically improved from mean 29.51 (SE 1.15) and mean 47.24 (SE 0.85) at baseline to mean 42.70 (SE 0.98) and mean 51.53 (SE 0.80) at week 18, respectively. Improvements were generally maintained to week 44 in part 2. Conclusion: Tofacitinib improved a range of PROs in patients with JIA, suggesting potential HRQoL benefits.