Background In Juvenile Idiopathic Arthritis (JIA) patients Etanercept is an effective therapeutic agent with an excellent safety profile. Currently it is licensed for treatment in polyarticular JIA patients over the age of 2 years.
Objectives To assess the efficacy and safety of Etanercept in JIA patients below the age of 2 years.
Methods The data base of the German BIKER registry documenting the treatment of 1499 JIA patients with Etanercept since 2001 has been used to identify patients who started treatment before the age of 2 years. Efficacy of treatment will be analyzed by achieving the ACRped30/50/70 criteria and single contributing domains. Safety analysis was performed on the basis of adverse event reports.
Results 7 patients (2 female) with a median age of 22.4 months (range 14-23 months) were identified. JIA category distribution varied from the total cohort: 4 had systemic JIA, 2 extended oligoarthritis and 1 seronegative polyarthitis. ANA were present in 2/6 pts, one pt had concomitant chronic uveitis. 3 received measles and 1 received varicella vaccination before start of Etanercept. All pts had received non-steroidal antirheumatic drugs, 6/7 methotrexate and 1 pt Cyclosporine A. The median initial weekly dosage of Etanercept was 0.8 mg/kg (0.66-1.05). 3 patients received once once weekly injections. Concomitant treatments were corticosteroids (n=2), methotrexate (n=7) and cyclosporine A (n=1).
At 3 months the ACR response levels were: ACR ped30, 86%; ACRped50, 71%; ACRped70, 57%; at 6 months: ACR ped30, 83%; ACRped50, 83%; ACRped70, 83%; at 12 months ACR ped30, 80%; ACRped50, 60%; ACRped70, 60%; at the last observation with Etanercept: ACR ped30, 71%; ACRped50, 71%; ACRped70, 71%. Furthermore, Etanercept provided clinically meaningful improvement for, Physician global assessment of disease activity; number of active joints, Childhood health assessment questionnaire disability index, ESR and CRP (Table).
Upon treatment, there were two adverse events in 2 patients; one pneumonia and one uveitis flare, one patient developed Hodgkin’s disease 5 years later. One patient discontinued Etanercept for inefficacy, 4 for reaching remission of their disease and one due to an adverse event (uveitis).
Conclusions In this small cohort of very young patients Etanercept proved safe and efficacious in the majority of children. The response as well as the tolerability was similar to that in older children. These preliminary results suggest that Etanercept therapy is a suitable and efficacious treatment option for patients with JIA under the age of two years. Further long term safety data are needed.
The German BIKER registry is supported by Pfizer Pharma
Disclosure of Interest G. Horneff Grant/Research support from: Pfizer, Abbott