Abstract
BackgroundSystemic juvenile idiopathic arthritis (sJIA) is a complex disease with dysregulation of the innate immune system driven by cytokines. A major role is ascribed to interleukin-1β (IL-1β), supporting the autoinflammatory character of the disease and offering an effective blocking mechanism for treatment. Here we present clinical practice data from the German AID-registry for patients treated with IL-1 inhibition (IL-1i).MethodsIn 2009 a clinical and research consortium (AID-Net) was established, including an online AID-registry. Patients with documented sJIA diagnosis were identified. Data for this retrospective IL-1i study were recorded by 17 centers. Response to treatment was evaluated according to Wallace criteria and additionally by an own classifying clinical response system.ResultsIn 6 years, 202 patients with confirmed sJIA were recorded in the AID-registry. Out of these, 111 children received therapy with Anakinra (ANA) (n = 84, 39 f) and/or Canakinumab (CANA) (n = 27, 15 f) at a median age of 8.7 y (range 0.6–19.1). During the first 12 months 75/111 (ANA 55, CANA 20) patients were evaluated according to Wallace criteria (achievement of inactive disease 28/55 and 17/20, remission over 6 months under medication 13/55 and 7/20 cases). Over the whole period of time, clinical response was preserved in the majority of patients (ANA 54/80, CANA 20/27). Arthritis mostly persisted in polyarticular (PA) courses. During treatment with IL-1i concomitant medication could be tapered in about 15%. IL-1i was discontinued in 59/111 patients. 45 (15) adverse events (AE)s in ANA (CANA) treated patients (19.7 (26.6) AE/100 ANA (CANA) exposure years, 95%CI: 14.4–26.4 (14.9–43.9)) were reported.ConclusionIn a large cohort of sJIA patients from Germany, we can confirm an overall favorable clinical response to both available IL-1 blocking agents. IL-1i was well tolerated with acceptable safety and effectiveness in a real-life clinical setting.
Highlights
Systemic juvenile idiopathic arthritis is a complex disease with dysregulation of the innate immune system driven by cytokines
SJIA is classified by the International League of Associations for Rheumatology (ILAR) as a category of juvenile idiopathic arthritis (JIA) and represents about 4,4% of 8096 JIA cases in the German National Pediatric Rheumatologic Database in 2017 [[3], unpublished data from German Rheumatism Research Centre (DRFZ)]
A total of 202/248 Systemic juvenile idiopathic arthritis (sJIA) children in the autoinflammatory disease (AID)-registry were enrolled in the study after exclusion of cases with secondary diagnoses or insufficient medical records
Summary
Systemic juvenile idiopathic arthritis (sJIA) is a complex disease with dysregulation of the innate immune system driven by cytokines. We present clinical practice data from the German AID-registry for patients treated with IL-1 inhibition (IL-1i). New strategies in diagnosing and managing sJIA as autoinflammatory disease (AID) have been recently published, and involve expensive cytokine-directed therapies against interleukin-1 (IL-1) and interleukin-6 (IL-6) [4, 7, 8]. Both treatment strategies are applied increasingly in sJIA cohorts since market approval of the available drugs. SJIA patients of AID-registry, treated with tocilizumab (TCZ) as anti-IL-6 blockade, reached inactive disease or remission after 1 year of treatment in 75% of patients [10]. Biological treatment has been proposed to foster a favorable long-term outcome in sJIA patients, leading to the hypothesis of a “window of opportunity” [14,15,16]
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