Functional Ability and Health-Related Quality of Life in Randomized Controlled Trials of Tocilizumab in Patients With Juvenile Idiopathic Arthritis

Objective To evaluate changes in health-related quality of life (HRQoL) and disability in children with systemic juvenile idiopathic arthritis (JIA) or polyarticular JIA treated with tocilizumab. Methods Secondary analyses of two double-blind, placebo-controlled trials of intravenous tocilizumab in children with active systemic JIA or polyarticular JIA were conducted. Patient-reported outcomes of disability (Childhood Health Assessment Questionnaire [C-HAQ]), HRQoL (Child Health Questionnaire Parent Form 50 [CHQ-P50], health concepts, physical summary score [CHQ-P50-PhS], psychosocial summary score [CHQ-P50-PsS]), pain, and well-being (100-mm visual analog scale [VAS]) were measured at weeks 0 and 12 for systemic JIA, weeks 16 and 40 for polyarticular JIA, and week 104 for both JIA subgroups. Results The trial included 112 patients with systemic JIA and 188 patients with polyarticular JIA. In patients with polyarticular JIA, the mean +/- SD C-HAQ score decreased from 1.39 +/- 0.74 at baseline to 0.67 +/- 0.65 at week 16 (P < 0.001). In patients with systemic JIA, the mean +/- SD CHQ-P50-PhS improved more with tocilizumab therapy than with placebo at week 12 (7.3 +/- 10.2 versus 2.4 +/- 10.6) (P < 0.05). Almost all mean CHQ-P50 health concept scores, CHQ-P50-PsS, and CHQ-P50-PhS improved (P <= 0.002) by week 104 for patients with systemic JIA. Patients with polyarticular JIA and patients with systemic JIA showed significant reductions in disability (mean +/- SD C-HAQ scores of -1.09 +/- 0.71 and -1.17 +/- 0.80, respectively), improvements in well-being (mean +/- SD well-being VAS scores of -43.76 +/- 26.61 and -51.53 +/- 23.57, respectively), and decreases in pain (mean +/- SD pain VAS scores of -41.56 +/- 31.06 and -51.26 +/- 26.79, respectively) (P < 0.001); in patients with polyarticular JIA and patients with systemic JIA who were treated with tocilizumab, 92.9% of polyarticular JIA patients and 96.8% of systemic JIA patients reported no more than minimal pain (a score of <= 35 mm on the VAS) at week 104. Conclusion Tocilizumab treatment was associated with significantly reduced disability and pain and improved HRQoL in patients with systemic JIA and polyarticular JIA.