Disability and Health-Related Quality of Life Outcomes in Patients with Systemic or Polyarticular Juvenile Idiopathic Arthritis Treated with Tocilizumab in Randomized Controlled Phase 3 Trials

Hermine Brunner¹, Chen Chen ¹, Alberto Martini ², Graciela Espada ², Rik Joos ², Jonathan Akikusa ², Jeffrey Chaitow ², Maria Luz Gámir Gámir ², Yukiko Kimura ³, Christoph Rietschel ², Daniel Siri ², Elzbieta Smolewska ², Heinrike Schmeling ¹, Diane Brown ¹, Fabrizio De Benedetti ⁴, Daniel J. Lovell ⁵, Bin Huang ¹ and Nicolino Ruperto ², ¹Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, ²Paediatric Rheumatology International Trials Organisation (PRINTO), Genoa, Italy, ³Joseph M Sanzari Children's Hospital, Hackensack University Medical Center, Cincinnati, OH, ⁴Bambino Gesù Children’s Hospital, Rome, Italy, ⁵Pediatric Rheumatology Collaborative Study Group (PRCSG), Cincinnati Children’s Hospital Medical Center, Cincinnati

Meeting: 2019 ACR/ARP Annual Meeting

Keywords: juvenile idiopathic arthritis (JIA), tocilizumab and quality of life

Session Information

Date: Monday, November 11, 2019

Title: 4M125: Pediatric Rheumatology: Outcomes & Quality of Life (1920–1925)

Session Type: ACR/ARP Abstract Session

Session Time: 4:30PM-6:00PM

Background/Purpose: Tocilizumab (TCZ) was approved for the treatment of systemic juvenile idiopathic arthritis (sJIA) and polyarticular juvenile idiopathic arthritis (pJIA) based on the results of 2 large phase 3 clinical trials.^1,2 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 in both trials. The objective of this post hoc analysis of data from both phase 3 trials of TCZ was to examine measures of disability and HRQOL in patients with sJIA or pJIA treated with TCZ for up to 2 years.

Methods: Eligible patients aged 2 to 17 years of age diagnosed with active (≥6 months) sJIA or pJIA according to International League of Associations for Rheumatology criteria received TCZ IV or placebo every 4 weeks (pJIA) or every 12 weeks (sJIA). For sJIA patients, 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, physician (MD) global assessment, and patient global assessment using analysis of variance adjusted for treatment group. Changes in CHAQ-DI overall and domain scores from baseline to 2 years were compared for sJIA and pJIA patients treated with TCZ using the unpaired t test; similarly, for sJIA patients treated with TCZ, changes in CHQ domain and summary scores between baseline and 2 years were assessed.

Results: sJIA patients experienced clinically relevant improvement of physical function (CHAQ-DI) and reduction in pain (pain-global). 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]). sJIA patients treated with TCZ had significantly improved socialization, behavior, mental health, and CHQ-psychosocial summary scores after 3 months compared with those receiving PBO (Table 1). Marked improvement in all CHAQ-DI domains over 2 years was observed with TCZ treatment in both sJIA and pJIA patients (Table 2); improvement rates in patient well-being (patient-global) were 87.7% in sJIA patients and 83.4% in pJIA patients. There was also significant improvement (p < 0.05) in most domains of HRQOL (CHQ-domain scores) in patients with sJIA; of note, sJIA 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).

Conclusion: TCZ treatment resulted in statistically significant and clinically relevant improvements in function and HRQOL in patients with sJIA or pJIA over 2 years.

References: 1. De Benedetti F et al. N Engl J Med 2012;367:2385-95. 2. Brunner HI et al. Ann Rheum Dis 2015;74;1110-7.

Disclosure: H. Brunner, ., 2, 5, 8, AbbVie, 5, AstraZeneca, 5, Bayer, 5, Bristol-Myers Squibb, 2, 5, EMD Serono, 5, Genentech, 2, 5, 8, GlaxoSmithKline, 5, Janssen, 5, Lilly, 5, Novartis, 5, 8, Pfizer, 2, 5, R-Pharm, 5, Sanofi, 5, UCB, 5; C. Chen, None; A. Martini, BMS, 9, GlaxoSmithKline, 9, Hoffman-La Roche, 9, Novartis, 5, 9, Pfizer, 5, 9, Sanofi Aventis, 5, 9, Schwarz Biosciences, 9, Abbott, 9, Francesco Angelini S.P.A., 9, Sobi, 9, Merck Serono, 9, Abbvie, 5, Boehringer, 5, Celgene, 5, CrescendoBio, 5, Janssen, 5, Medimmune, 5, NovoNordisk, 5, Vertex, 5, Servier, 5; G. Espada, None; R. Joos, None; J. Akikusa, None; J. Chaitow, None; M. Gámir Gámir, None; Y. Kimura, Novartis, 5, Sobi, 5; C. Rietschel, None; D. Siri, None; E. Smolewska, None; H. Schmeling, F. Hoffmann-La Roche Ltd, 2, Janssen, 2, Pfizer, 2, UCB Biosciences GmbH, 2; D. Brown, None; F. De Benedetti, AbbVie, 2, BMS, 2, Novartis, 2, Novartis, Novimmune, Hoffmann- La Roche, SOBI, AbbVie, Pfizer, 2, Novimmune, 2, Pfizer, 2, Roche, 2, Sanofi, 2, Sobi, 2, Swedish Orphan Biovitrum, 2, UCB, 2; D. Lovell, Abbott, 5, 9, AbbVie, 5, 9, Amgen, 5, 9, AstraZeneca, 5, Astra-Zeneca Pharm, 5, Biogen, 5, Boehringer Ingelheim, 5, Boeringher Ingelheim, 5, Bristol-Myers Squibb, 5, 9, Celgene, 5, Forest Research, 9, Forest Research Institute, 5, Genentech, 5, 8, GlaxoSmithKline, 5, Hoffmann-La Roche, 5, 9, Horizon, 5, Jannsen, 5, Janssen, 5, 9, Johnson & Johnson, 5, Novartis, 5, 9, Pfizer, 5, 9, Roche, 5, 9, Takeda, 5, 9, UBC, 5, Wyeth Pharm, 5, 8; B. Huang, None; N. Ruperto, AbbVie, 5, 8, Abbvie, 8, Ablynx, 5, 8, Ablynx, AbbVie, Astrazeneca-Medimmune, Biogen, Boehringer, Bristol Myers and Squibb, Eli-Lilly, EMD Serono, Glaxo Smith and Kline, 8, AstraZeneca-Medimmune, 8, Astrazeneca-Medimmune, 8, AstraZeneca-MedImmune, 5, 8, Biogen, 5, 8, BMS, Eli-Lilly, GlaxoSmithKline, F Hoffmann-La Roche, Janssen, Novartis, Pfizer, Sobi., 2, Boehringer, 8, Boehringer Ingelheim, 5, 8, Boeringher Ingelheim, 8, Bristol-Myers Squibb, 2, 5, 8, Eli Lilly, 2, 5, 8, Eli-Lilly, 8, EMD Serono, 5, 8, F Hoffmann-La Roche, 2, 5, 8, GlaxoSmithKline, 2, 5, 8, Hoffmann-La Roche, 8, Hoffmann-La Roche, Janssen, Merck, Novartis, Pfizer, R-Pharma, SanofiServier, Sinergie, Sobi and Takeda, 8, Janssen, 2, 5, 8, Merck, 5, 8, Novartis, 2, 5, 8, Pfizer, 2, 5, 8, R-Pharma, 5, 8, SanofiServier, 5, 8, Sinergie, 5, 8, Sobi, 2, 5, 8, 9, Takeda, 5, 8.

To cite this abstract in AMA style:

Brunner H, Chen C, Martini A, Espada G, Joos R, Akikusa J, Chaitow J, Gámir Gámir M, Kimura Y, Rietschel C, Siri D, Smolewska E, Schmeling H, Brown D, De Benedetti F, Lovell D, Huang B, Ruperto N. Disability and Health-Related Quality of Life Outcomes in Patients with Systemic or Polyarticular Juvenile Idiopathic Arthritis Treated with Tocilizumab in Randomized Controlled Phase 3 Trials [abstract]. Arthritis Rheumatol. 2019; 71 (suppl 10). https://acrabstracts.org/abstract/disability-and-health-related-quality-of-life-outcomes-in-patients-with-systemic-or-polyarticular-juvenile-idiopathic-arthritis-treated-with-tocilizumab-in-randomized-controlled-phase-3-trials/. Accessed .

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