O53. Efficacy and Safety of Tocilizumab in Polyarticular-Course Juvenile Idiopathic Arthritis: 2 Year Data from Cherish

Abstract

Background: Efficacy and safety of tocilizumab (TCZ), an IL‐6 receptor inhibitor, were previously demonstrated at week 40 of CHERISH, a phase 3 trial in patients (pts) with polyarticular‐course JIA (pcJIA). This analysis investigates the efficacy and safety of TCZ over 104 weeks of treatment in pcJIA. Methods: Pts aged 2‐17 years with active pcJIA who failed MTX received open‐label (OL) TCZ [body weight (BW) <30 kg: 8 mg/kg (n=119); BW <30 kg: pts randomly assigned to 8 mg/kg (n=34) or 10 mg/kg (n=35)] every 4 weeks for 16 weeks. At week 16, eligible pts (JIA ACR30 response) entered a 24‐week randomized [1:1 placebo or continuation of TCZ)], double‐blind withdrawal period (DBWP) to evaluate the primary endpoint (JIA ACR30 flare relative to week 16). Pts with JIA ACR30 flare or who completed the DBWP entered an OLextension study through week 104. Results: 188 pts entered the OL lead‐in period, 166 entered the DBWP, 160 entered the OL‐extension, 155 completed 104 weeks. In patients who received TCZ throughout the study, JIA ACR responses and improvement in JIA ACR core components (Table 1) were maintained through week 104. The safety population comprised 188 patients with 307 patient‐years (PY). Rates/100 PY of adverse events (AEs) and serious AEs (SAEs) were 406.5 and 11.1, respectively; infections were the most common AE (151.4) and SAE (5.2). Alanine aminotransferase and aspartate aminotransferase elevations 3 upper limit of normal occurred in 6.4% and 2.7% of patients, respectively. Grade 3 neutropenia and grade 2/3/4 thrombocytopenia occurred in 5.9% and 1.6% of patients, respectively. Low‐density lipoprotein cholesterol >110 mg/dl occurred in 16.2%. Conclusion: Efficacy of TCZ was maintained through 2 years of treatment in patients with pcJIA, with no change in safety profile from that reported previously. (Table presented).