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)

Pierre Quartier, Florence Allantaz, Rolando Cimaz, Pascal Pillet, Claude Messiaen, Christophe Bardin, Xavier Bossuyt, Anne Boutten, Jacques Bienvenu, Agnes Duquesne, Olivier Richer, Damien J. Chaussabel, Agnes Mogenet, Jacques Banchereau, Jean Marc Treluyer, Paul Landais, Virginia Pascual

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Abstract

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.

Original languageEnglish
Pages (from-to)747-754
Number of pages8
JournalAnnals of the Rheumatic Diseases
Volume70
Issue number5
DOIs
Publication statusPublished - May 2011
Externally publishedYes

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ASJC Scopus subject areas

  • Rheumatology
  • Immunology
  • Biochemistry, Genetics and Molecular Biology(all)
  • Immunology and Allergy

Cite this

Quartier, P., Allantaz, F., Cimaz, R., Pillet, P., Messiaen, C., Bardin, C., Bossuyt, X., Boutten, A., Bienvenu, J., Duquesne, A., Richer, O., Chaussabel, D. J., Mogenet, A., Banchereau, J., Treluyer, J. M., Landais, P., & Pascual, V. (2011). 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). Annals of the Rheumatic Diseases, 70(5), 747-754. https://doi.org/10.1136/ard.2010.134254