Background Patient’s quality of life is markedly affected by the persistent and disabling disease features of systemic juvenile idiopathic arthritis (SJIA). We evaluated the direct impact of the disease on patients’ well being and the improvements observed following treatment with a single dose of canakinumab (CAN) using a variety of patient reported outcomes (PROs).
Objectives To assess improvement in Health-Related Quality of Life (HRQoL) with CAN vs placebo for the treatment of SJIA patients.
Methods In a 4-week, randomized, double blind, single-dose study (β-SPECIFIC 1), 84 patients with active systemic features at enrollment were randomized 1:1 to receive a single dose of CAN (4mg/kg) or placebo sc on Day 1. The primary endpoint was proportion of patients achieving an adapted ACRpedi 30 criteria (plus absence of fever) at Day 15. Secondary endpoints included change in HRQoL and disability over time, pain intensity (0-100 mm VAS) and safety. Both concepts were measured by CHQ-PF50 and CHAQ© respectively.
Results 84 patients (2-18 yrs) were randomized to CAN (n=43) or placebo (n=41). High discontinuation rate in the placebo group (90.2%) was due to unsatisfactory therapeutic response vs CAN group (14%). At baseline (BL), the median CHQ-PF50 physical and psychosocial scores for all patients (5-18 yrs, n=28) were 19.2 and 40.0 for CAN and 18.1 and 44.9 for placebo. Median baseline CHAQ score was 1.6 for CAN and 1.5 for placebo. At BL, mean pain intensity was 69.7mm for CAN and 60.9mm for placebo. CAN provided statistically significant higher improvement in CHQ-PF50 physical and psychosocial scores compared to placebo (Table). The median improvement in the CHAQ disability score achieved with CAN both at Days 15 and 29 exceeded a previously established minimal clinical important difference (MCID) cited to be -0.19 by at least a factor of 5. Least square means in overall pain intensity were significantly lower in the CAN vs placebo group at Days 15 and 29 (both, p<0.0001). 55.8% patients in the CAN vs 39.0% placebo group had AEs with the most frequently reported AEs being upper respiratory tract infections. 4 patients had serious AEs, 2 in each treatment group.
Conclusions A single dose of canakinumab demonstrated significant and rapid improvement in HRQoL and baseline disability of SJIA patients. Such benefits will be of high importance to patients and payers.
Disclosure of Interest N. Wulffraat: None Declared, T. Kallinich: None Declared, L. McCann: None Declared, R. Brik: None Declared, L. Rutkowska-Sak: None Declared, M. Ferrandiz: None Declared, K. Lheritier Shareholder of: Novartis, Employee of: Novartis, D. Kim Shareholder of: Novartis, Employee of: Novartis, A. Gnanasakthy Employee of: Novartis, H. Ozdogan Speakers Bureau: Novartis