Abstract
BackgroundSystemic juvenile idiopathic arthritis (sJIA) is a complex disease with an autoinflammatory component of unknown etiology related to the innate immune system. A major role in the pathogenesis has been ascribed to proinflammatory cytokines like interleukin-6 (IL-6), and effective drugs inhibiting their signaling are being developed. This study evaluates sJIA patients treated with the IL-6 inhibitor tocilizumab (TCZ) concerning clinical response rate, disease course and adverse effects in a real-life clinical setting.MethodsIn 2009 a clinical and research consortium was established, including an online registry for autoinflammatory diseases (AID) (https://aid-register.de). Data for this retrospective TCZ study were documented by 13 centers.ResultsFrom 7/2009 to 4/2014, 200 patients with sJIA were recorded in the AID-registry. Out of these, 46 (19 m, 27 f, age 1–18 years) received therapy with TCZ. Long term treatment (median 23 months) has been documented in 24/46 patients who were evaluated according to Wallace criteria (active disease 6/24, inactive disease 5/24, remission 13/24 cases). Under observation co-medication were used in 40/46 cases. Adverse events were reported in 11/46 patients. The clinical response rate (no clinical manifestation, no increased inflammation parameters) within the first 12 weeks of treatment was calculated to be 35%.ConclusionOut of 200 sJIA children reported in the German AID-registry, 46 were treated with TCZ, showing a clinical response rate of 35% during the first 12 weeks, and inactive disease and/or remission under medication in 75% after one year. Adverse events were seen in 24% and severe adverse events in 4%.Trial registrationThe AID-Registry is funded by the BMBF (01GM08104, 01GM1112D, 01GM1512D).
Highlights
Systemic juvenile idiopathic arthritis is a complex disease with an autoinflammatory component of unknown etiology related to the innate immune system
We present the first followup results of the autoinflammatory diseases (AID)-registry work concerning clinical response rates, achievement of inactive disease and remission (Wallace criteria), disease courses, inflammatory parameters, outcome, concomitant medication and adverse effects from 46 out of 200 Systemic juvenile idiopathic arthritis (sJIA) patients treated with TCZ in Germany in a real-life clinical setting
From other East European (Bulgaria, Albania) and Asian countries and 8/46 (17%) of unknown or mixed ethnicity. 34/46 (74%) patients fulfilled International League of Associations for Rheumatology (ILAR) classification criteria, 12/46 (26%) patients did not fulfill ILAR criteria but other definitions for sJIA and diagnosis was confirmed by pediatric rheumatologists [3, 18,19,20]
Summary
Systemic juvenile idiopathic arthritis (sJIA) is a complex disease with an autoinflammatory component of unknown etiology related to the innate immune system. Systemic juvenile idiopathic arthritis (sJIA) is a complex disease with an autoinflammatory component of unknown origin. SJIA is classified by the International League of Associations for Rheumatology (ILAR) as a subtype of juvenile idiopathic arthritis (JIA) and represents about 10–20% of all JIA in the Caucasian population [2]. The ILAR classification criteria for sJIA have been criticized in the last years. Various initiatives developed new criteria for pediatric population (CARRA, PRINTO, GKJR) [3,4,5]. SJIA is associated with erosive arthritis, growth retardation, cardiovascular and pulmonary morbidity as well as amyloidosis. The prevalence of amyloidosis was approximately 1–2% until the 1990s and decreased continuously in the last years [7]
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