FRI0572 DISABILITY AND HEALTH-RELATED QUALITY OF LIFE OUTCOMES IN PATIENTS WITH SYSTEMIC JUVENILE IDIOPATHIC ARTHRITIS TREATED WITH TOCILIZUMAB IN A PHASE 3 RANDOMIZED CONTROLLED TRIAL

Background Tocilizumab (TCZ) intravenous (IV) formulation was approved for the treatment of patients with systemic juvenile idiopathic arthritis (sJIA) based on the results of a large phase 3 clinical trial. Physical function, measured by the Childhood Health Assessment Questionnaire–Disability Index (CHAQ-DI), and health-related quality of life (HRQOL), measured by the Child Health Questionnaire (CHQ), were evaluated. Objectives To examine measures of disability and HRQOL in patients with sJIA treated with TCZ IV for up to 2 years in post hoc analysis of data from the phase 3 trial of TCZ. Methods Changes within 3 months of treatment initiation with TCZ (baseline) were compared between TCZ- and placebo (PBO)–treated patients using CHAQ-DI, pain global assessment (Pain-GA), physician global assessment, and patient global assessment (Pt-GA) using analysis of variance adjusted for treatment group. Changes in CHAQ-DI overall and domain scores and changes in CHQ domain and summary scores from baseline to 2 years were compared for patients treated with TCZ using the unpaired t test. Results Patients with sJIA experienced clinically relevant improvement in physical function (CHAQ-DI) and reduction of pain (Pain-GA). Mean (SD) CHAQ-DI scores for patients treated with PBO and TCZ were 1.7 (0.8) for both groups at baseline and 1.2 (1.0) and 0.9 (0.8), respectively, at week 12 (week 12 mean difference, –0.2; median [range] change from baseline to week 12 difference, –0.3 [–0.6 to 0.0]). These patients also had significantly improved CHQ socialization, behavior, mental health, and psychosocial summary scores after 3 months compared with those receiving PBO (Figure 1). Improvement in all CHAQ-DI domains over 2 years was observed with TCZ treatment (Figure 2); improvement rates in patient well-being (Pt-GA) were 87.7%. Similar improvements in physical function were observed in patients with polyarticular JIA in the phase 3 trial that led to approval of TCZ IV in these patients (not shown). There was also significant improvement (p Conclusion Two years of TCZ treatment resulted in statistically significant and clinically relevant improvements in function and HRQOL in patients with sJIA. Disclosure of Interests Nicolino Ruperto Grant/research support from: The Gaslini Hospital, where NR works as full-time public employee, has received contributions (> 10.000 USD each) from the following industries in the last 3 years: BMS, Eli-Lilly, GlaxoSmithKline, F Hoffmann-La Roche, Janssen, Novartis, Pfizer, Sobi. This funding has been reinvested for the research activities of the hospital in a fully independent manner, without any commitment with third parties., Consultant for: Received honoraria for consultancies or speaker bureaus (