Abstract
Systemic onset juvenile idiopathic arthritis (SoJIA) is a rare inflammatory disorder. It is the severest form of juvenile idiopathic arthritis, and complications occur most commonly in this type. Non-responsiveness to standard therapy with corticosteroids and disease-modifying antirheumatic drugs is not uncommon. Interleukin-1 beta (Il-1β) has been shown to be a main contributor to the pathogenesis of SoJIA. Anakinra, a recombinant Il1β receptor antagonist, was shown to be effective in small cohorts of therapy-resistant adult and pediatric still's patients. This study aimed to evaluate the real-world efficacy, steroid-sparing effect, and safety profile of anakinra in patients with SoJIA at a tertiary care center in Libya. A retrospective case series was conducted on patients with SoJIA treated with anakinra at the Tripoli Children's Hospital between 2010 and 2017. Data on demographic characteristics, disease activity, corticosteroid dosage, concomitant medications, and adverse events were collected at baseline and at 1-, 3-, 6-, and 12-month post-treatment. 13 patients were treated with anakinra with a mean age of 9.4±4.6 years at anakinra initiation and a female-to-male ratio of 2: 1. All patients were on corticosteroids and 92.3% on methotrexate at treatment initiation. The proportion of patients achieving inactive disease (Jadas 10=0) increased over time. A marked steroid-sparing effect was observed: the number of patients requiring high-dose steroids (>0.5 mg/kg/day) decreased from 100% at baseline to 7.6% at 12 months, and 53.8% successfully discontinued corticosteroids entirely. All patients experienced injection site reaction, and macrophage activation syndrome occurred in 15.4% as a side effect after treatment initiation. But no severe infections or fatalities occurred. Reasons for discontinuation included remission (46.1%), drug unavailability (23.0%), inefficacy (15.3%), and side effects (15.3%). Anakinra demonstrated significant efficacy in inducing rapid disease control and reducing corticosteroid dependence in patients with refractory SoJIA, with a manageable safety profile.