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

1. Tofacitinib significantly improves patient-reported outcomes compared to placebo in the management of juvenile idiopathic arthritis (JIA).

Evidence Rating Level: 2 (Good)

Tofacitinib is an oral Janus kinase (JAK) inhibitor for the treatment of JIA. Apart from clinical and biochemical markers, patient-reported outcomes (PRO; e.g. physical function, physical and psychological well-being, and pain levels) are key determinants of efficacy. This is a post-hoc analysis of a phase 3, 44-week, randomized, double-blind, placebo-controlled trial. In the open-label run-in phase (part 1, 18 weeks), patients received tofacitinib. During the double-blind phase (part 2, 26 weeks), patients were randomized to continue tofacitinib or switch to placebo for up to an additional 26 weeks. PRO was assessed with the Childhood Health Assessment Questionnaire (CHAQ) and the Child Health Questionnaire (CHQ). 225 patients (median [IQR] age, 13.0 [9.0-15.0] years; 75.1% female) were enrolled in part 1, 173 of which were randomized in part 2. At week 18, 48.0% of patients achieved minimal clinically important differences (MCID) in CHAQ-DI (score reduction of ≥0.188). The proportion of patients with no disability (CHAQ-disability index = 0) increased from 12.9% (SE 2.2) at baseline to 28.0% (SE 3.0) at week 18. The proportion of patients achieving minimal arthritis pain (CHAQ-Discomfort Index score of ≤0.35) increased from 4.4% (SE 1.4) at baseline to 15.6% (SE 2.4) at week 18. In part 2, a higher proportion of patients receiving tofacitinib achieved no disability compared with patients receiving placebo (38.6% vs. 23.5%; P < 0.05). A higher proportion of patients achieved minimal arthritis pain in the tofacitinib group than in the placebo group (26.1% vs. 11.8%; P < 0.05). Although the efficacy of tofacitinib versus placebo in the treatment of JIA has previously been shown, these results capture aspects of disease that may not be captured by clinical or biochemical markers. Overall, tofacitinib significantly improves patient-reported quality of life compared to placebo.

Click here to read this study in Arthritis & Rheumatology

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