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
Authors: Nicolino Ruperto, Chen Chen, Alberto Martini, Graciela Espada, Rik Joos, Jonathan Akikusa, Jeffrey Chaitow, Gámir Gámir Maria Luz, Yukiko Kimura, Christoph Rietschel, Daniel Siri, Elzbieta Smolewska, Heinrike Schmeling, Diane Brown, Fabrizio De Benedetti, Daniel J. Lovell, Bin Huang, Hermine Brunner
Journal: Annals of the Rheumatic Diseases (2019)

<h3>Background</h3> 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. <h3>Objectives</h3> 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. <h3>Methods</h3> 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 <i>t</i> test. <h3>Results</h3> 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 (<i>p</i>&lt;0.05) in most domains of HRQOL (CHQ domain scores) in patients with sJIA; of note, patients experienced marked improvement in mean scores from baseline to week 104 for pain/discomfort (31.7 to 75.3), self-esteem (61.0 to 76.0), mental health (62.1 to 76.8), and social limitation-emotional (52.6 to 86.2). <h3>Conclusion</h3> Two years of TCZ treatment resulted in statistically significant and clinically relevant improvements in function and HRQOL in patients with sJIA. <h3>Disclosure of Interests</h3> Nicolino Ruperto Grant/research support from: The Gaslini Hospital, where NR works as full-time public employee, has received contributions (&gt; 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 (&lt; 10.000 USD each) from the following pharmaceutical companies in the past 3 years: Ablynx, AbbVie, Astrazeneca-Medimmune, Biogen, Boehringer, Bristol-Myers Squibb, Eli-Lilly, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, SanofiServier, Sinergie, Sobi and Takeda., Speakers bureau: Received honoraria for consultancies or speaker bureaus (&lt; 10.000 USD each) from the following pharmaceutical companies in the past 3 years: Ablynx, AbbVie, Astrazeneca-Medimmune, Biogen, Boehringer, Bristol-Myers Squibb, Eli-Lilly, EMD Serono, GlaxoSmithKline, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, SanofiServier, Sinergie, Sobi and Takeda., Chen Chen: None declared, Alberto Martini: None declared, Graciela Espada: None declared, Rik Joos: None declared, Jonathan Akikusa: None declared, Jeffrey Chaitow: None declared, Maria Luz Gámir Gámir: None declared, Yukiko Kimura Consultant for: Novartis, SOBI, Christoph Rietschel: None declared, Daniel Siri: None declared, Elzbieta Smolewska: None declared, Heinrike Schmeling Grant/research support from: F. Hoffmann-La Roche Ltd, Diane Brown: None declared, Fabrizio De Benedetti Grant/research support from: Abbvie, SOBI, Novimmune, Roche, Novartis, Sanofi, Pfizer, Daniel J Lovell Consultant for: Consulting fees and/or honoraria from Astra Zeneca, Wyeth Pharma, Amgen, Abbott, Pfizer, F. Hoffmann-La Roche, Novartis, UBC, Takeda, GSK, Boehringer, and Celgene, Bin Huang: None declared, Hermine Brunner Grant/research support from: Bristol-Myers Squibb, Pfizer, Consultant for: Pfizer, Bristol-Myers Squibb, Janssen, Novartis, Lilly, Roche, GlaxoSmithKline, Sanofi, Speakers bureau: Novartis, Roche