EULAR 2009: Etanercept Safe, Effective in Young Children With Juvenile Idiopathic Arthritis

Alice Goodman

June 16, 2009

June 16, 2009 (Copenhagen, Denmark) — Etanercept appears to be safe and effective in children younger than 4 years with juvenile idiopathic arthritis (JIA), a group for whom data are lacking with this treatment, according to results from an open-label pilot study. Children in the study were followed at a large tertiary-care hospital and appeared to have more severe JIA than those seen in general clinical practice.

"In patients under the age of 4 years, etanercept had similar efficacy in reducing disease and maintaining disease remission [as older children], with a similar safety profile. However, we need larger randomized studies to confirm this observation," said Antonella Insalaco, MD, from Ospedale Pediatrico Bambino Gesù in Rome, Italy, who presented the findings here at EULAR 2009: The Annual European Congress of Rheumatology.

"It's absolutely necessary to characterize the role of etanercept in a double-blind controlled study, which we are planning to do in the future," said lead author Claudia Bracaglia, MD, a pediatric rheumatologist at Ospedale Pediatrico Bambino Gesù.

Etanercept is not approved in Europe for the treatment of JIA patients younger than 4 years, she explained. This drug has shown safety and efficacy in clinical trials of older children with JIA, with no increase in serious adverse events after as long as 8 years of follow-up, Dr. Insalaco explained.

The study involved 33 patients younger than 4 years (24 girls and 9 boys) who had JIA and who did not respond to methotrexate. Mean disease duration was 12 months. Patients were treated with etanercept (dose range, 0.8 to 1.0 mg/kg) for a mean of 23 months (range, 6 to 86 months). Eight patients (24%) presented with systemic onset of JIA, 18 (58%) presented with oligoarticular onset (5 persistent, 13 extended), 6 presented with polyarticular onset (rheumatoid-factor negative), and 1 (3%) presented with psoriatic arthritis.

All patients were taking concomitant drugs: 31 were taking methotrexate and 2 were taking cyclosporine. After 6 months of treatment, 82% of patients achieved at least a 30% improvement on the American College of Rheumatology scoring system (ACR30), 63% achieved at least a 50% improvement (ACR50), and 42% achieved at least a 70% improvement (ACR70). At the last follow-up, response rates on these measures were 85%, 85%, and 73%, respectively. Five patients (5%) stopped taking etanercept because of a lack of efficacy after 5 months; 1 was switched to a different tumor necrosis factor (TNF)-alpha inhibitor and 4 were switched to an interleukin-1 blocker.

No major adverse events were reported in 82% of the study population. Four patients (12%) had a major adverse treatment-related event: 3 varicella zoster virus infections (1 requiring hospitalization for necrotizing fasciitis) and 1 cytomegalovirus infection. Dr. Insalaco said that now all patients are routinely vaccinated for varicella zoster virus prior to treatment with methotrexate or TNF-alpha agents. No patient treated with etanercept developed tuberculosis, other viral infection, or malignancy.

These data in younger children compare favorably with historic data in children 5 to 20 years with JIA treated with etanercept, she said.

Aggressive Treatment Questioned

The session moderators — Silvia Magni-Manzoni, MD, from Fondazione San Matteo in Pavia, Italy, and Freddy Karup Pedersen, MD, from University Hospital Rigshospitalet in Copenhagen, Denmark — agreed that this represented a larger cohort of JIA children with more severe disease than would be expected at a regular clinic.

Both physicians said that it was surprising how aggressively the younger children in this study were treated. "In many places, [nonsteroidal anti-inflammatory drugs] are used, especially for children with oligoarthritis. It is not routine to use methotrexate and TNF-alpha inhibitors in such young children," Dr. Pedersen stated.

The study did not receive commercial support. Dr. Insalaco, Dr. Magni-Manzoni, and Dr. Pedersen have disclosed no relevant financial relationships.

EULAR 2009: The Annual European Congress of Rheumatology. Abstract OP-0137. Presented June 11, 2009.


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