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Efficacy, safety and effect on gene expression profiling of anakinra in systemic-onset juvenile idiopathic arthritis: final results of a randomised, double-blind, placebo-controlled trial (ANAJIS)

Purpose

To investigate efficacy and safety of anakinra in systemic-onset juvenile idiopathic arthritis patients. To assess treatment effect on gene expression profiling, immune response to anti-pneumococcal Pneumo23 vaccine, serum amyloid A level, serum ferritin level and the percentage of glycosylated ferritin.

Methods

Multicenter randomized double-blind trial. The primary objective was to compare the efficacy of a one-month treatment with anakinra to a placebo between 2 groups of 12 patients each. Response was defined by 30% improvement of pediatric ACR core-set criteria for JIA, resolution of fever and systemic symptoms and normalization or a decrease of at least 50% of both CRP and first hour ESR compared to baseline. Intention-to-treat analysis. Secondary objectives included tolerance and efficacy assessment over 12 months and treatment effect on blood gene expression profiling.

Results

At one month, there was a significant difference in the response rate between patients treated with anakinra (8/12) and placebo (1/12). During the double-blind phase, the number of adverse events, mainly pain to injections, was similar between both groups. Ten patients from the placebo group switched to anakinra at Month 1 and 9 were responders at month 2. Eight patients discontinued anakinra before Month 12: painful injections during the double-blind phase (2 patients, both on placebo), ileocolic symptoms leading to the diagnosis of Crohn's disease (1 patient), transient hepatic cytolysis (one case), lack of efficacy or a disease flare (4 cases). Gene expression profile analyses showed a set of gene pathways dysregulated in SOJIA whose expression dramatically changed upon anakinra treatment.

Author information

Correspondence to P Quartier.

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Open Access This article is published under license to BioMed Central Ltd. This is an Open Access article is distributed under the terms of the Creative Commons Attribution 2.0 International License (https://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Keywords

  • Ferritin
  • Gene Expression Profile
  • Anakinra
  • Serum Ferritin Level
  • Juvenile Idiopathic Arthritis Patient