PT  - JOURNAL ARTICLE
AU  - Pierre Quartier
AU  - Florence Allantaz
AU  - Rolando Cimaz
AU  - Pascal Pillet
AU  - Claude Messiaen
AU  - Christophe Bardin
AU  - Xavier Bossuyt
AU  - Anne Boutten
AU  - Jacques Bienvenu
AU  - Agnes Duquesne
AU  - Olivier Richer
AU  - Damien Chaussabel
AU  - Agnes Mogenet
AU  - Jacques Banchereau
AU  - Jean-Marc Treluyer
AU  - Paul Landais
AU  - Virginia Pascual
TI  - A multicentre, randomised, double-blind, placebo-controlled trial with the interleukin-1 receptor antagonist anakinra in patients with systemic-onset juvenile idiopathic arthritis (ANAJIS trial)
AID  - 10.1136/ard.2010.134254
DP  - 2011 May 01
TA  - Annals of the Rheumatic Diseases
PG  - 747--754
VI  - 70
IP  - 5
4099  - http://ard.bmj.com/content/70/5/747.short
4100  - http://ard.bmj.com/content/70/5/747.full
SO  - Ann Rheum Dis2011 May 01; 70
AB  - Objectives To assess the efficacy of the interleukin 1 receptor antagonist anakinra in systemic-onset juvenile idiopathic arthritis (SJIA). Methods A multicentre, randomised, double-blind, placebo-controlled trial was conducted. The primary objective was to compare the efficacy of a 1-month treatment with anakinra (2 mg/kg subcutaneous daily, maximum 100 mg) with a placebo between two groups each with 12 patients with SJIA. Response was defined by a 30% improvement of the paediatric American College of Rheumatology criteria for JIA, resolution of systemic symptoms and a decrease of at least 50% of both C-reactive protein and erythrocyte sedimentation rate compared with baseline. After month 1 (M1), patients taking placebo were switched to anakinra. Secondary objectives included tolerance and efficacy assessment for 12 months, and analyses of treatment effect on blood gene expression profiling. Results At M1, 8/12 responders were receiving anakinra and 1 responder receiving placebo (p=0.003). Ten patients from the placebo group switched to anakinra; nine were responders at M2. Between M1 and M12, six patients stopped treatment owing to an adverse event (n=2), lack of efficacy (n=2) or a disease flare (n=2). Blood gene expression profiling at enrolment and at 6 months' follow-up showed one set of dysregulated genes that reverted to normal values in the clinical responders and a different set, including interferon (IFN)-inducible genes, that was induced by anakinra. Conclusions Anakinra treatment is effective in SJIA, at least in the short term. It is associated with normalisation of blood gene expression profiles in clinical responders and induces a de novo IFN signature. Trial Registration Number: NCT00339157.