PT  - JOURNAL ARTICLE
AU  - Brunner, Hermine I
AU  - Ruperto, Nicolino
AU  - Zuber, Zbigniew
AU  - Keane, Caroline
AU  - Harari, Olivier
AU  - Kenwright, Andrew
AU  - Lu, Peng
AU  - Cuttica, Ruben
AU  - Keltsev, Vladimir
AU  - Xavier, Ricardo M
AU  - Calvo, Inmaculada
AU  - Nikishina, Irina
AU  - Rubio-Pérez, Nadina
AU  - Alexeeva, Ekaterina
AU  - Chasnyk, Vyacheslav
AU  - Horneff, Gerd
AU  - Opoka-Winiarska, Violetta
AU  - Quartier, Pierre
AU  - Silva, Clovis A
AU  - Silverman, Earl
AU  - Spindler, Alberto
AU  - Baildam, Eileen
AU  - Gámir, M Luz
AU  - Martin, Alan
AU  - Rietschel, Christoph
AU  - Siri, Daniel
AU  - Smolewska, Elzbieta
AU  - Lovell, Daniel
AU  - Martini, Alberto
AU  - De Benedetti, Fabrizio
AU - for the Paediatric Rheumatology International Trials Organisation (PRINTO) and the Pediatric Rheumatology Collaborative Study Group (PRCSG)
TI  - Efficacy and safety of tocilizumab in patients with polyarticular-course juvenile idiopathic arthritis: results from a phase 3, randomised, double-blind withdrawal trial
AID  - 10.1136/annrheumdis-2014-205351
DP  - 2015 Jun 01
TA  - Annals of the Rheumatic Diseases
PG  - 1110--1117
VI  - 74
IP  - 6
4099  - http://ard.bmj.com/content/74/6/1110.short
4100  - http://ard.bmj.com/content/74/6/1110.full
SO  - Ann Rheum Dis2015 Jun 01; 74
AB  - Objective To evaluate the interleukin-6 receptor inhibitor tocilizumab for the treatment of patients with polyarticular-course juvenile idiopathic arthritis (pcJIA).Methods This three-part, randomised, placebo-controlled, double-blind withdrawal study (NCT00988221) included patients who had active pcJIA for ≥6 months and inadequate responses to methotrexate. During part 1, patients received open-label tocilizumab every 4 weeks (8 or 10 mg/kg for body weight (BW) <30 kg; 8 mg/kg for BW ≥30 kg). At week 16, patients with ≥JIA-American College of Rheumatology (ACR) 30 improvement entered the 24-week, double-blind part 2 after randomisation 1:1 to placebo or tocilizumab (stratified by methotrexate and steroid background therapy) for evaluation of the primary end point: JIA flare, compared with week 16. Patients flaring or completing part 2 received open-label tocilizumab.Results In part 1, 188 patients received tocilizumab (<30 kg: 10 mg/kg (n=35) or 8 mg/kg (n=34); ≥30 kg: n=119). In part 2, 163 patients received tocilizumab (n=82) or placebo (n=81). JIA flare occurred in 48.1% of patients on placebo versus 25.6% continuing tocilizumab (difference in means adjusted for stratification: −0.21; 95% CI −0.35 to −0.08; p=0.0024). At the end of part 2, 64.6% and 45.1% of patients receiving tocilizumab had JIA-ACR70 and JIA-ACR90 responses, respectively. Rates/100 patient-years (PY) of adverse events (AEs) and serious AEs (SAEs) were 480 and 12.5, respectively; infections were the most common SAE (4.9/100 PY).Conclusions Tocilizumab treatment results in significant improvement, maintained over time, of pcJIA signs and symptoms and has a safety profile consistent with that for adults with rheumatoid arthritis.Trial registration number: NCT00988221.