Ruperto N et al. (2008) Abatacept in children with juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled withdrawal trial. Lancet 372: 383–391

Methotrexate, a DMARD used to treat rheumatoid arthritis, is also suitable for treating patients with juvenile idiopathic arthritis (JIA). Children who fail to respond to methotrexate can be treated with anti-tumor necrosis factor, although some do not respond or are intolerant to this line of therapy. Studies have shown that, in adults with rheumatoid arthritis, treatment with abatacept, a selective T-cell inhibitor, improves the condition. Ruperto and colleagues, therefore, investigated the effects of abatacept in children with JIA refractory to standard treatment.

Patients with JIA (n = 190, aged 6–17 years), recruited from medical centers in Europe, Latin America and the USA, received five doses of 10 mg/kg abatacept (30 min intravenous infusion) during an open-label period of 4 months. Those who responded positively were randomly assigned to receive either further treatment with abatacept (n = 60) or placebo (n = 62) at the point of randomization and every 28 days thereafter for 6 months, or until a disease flare-up. More patients in the abatacept group had improved by at least 50% compared with controls (77% vs 52%, P = 0.0071). More flare-ups occurred in patients who were given placebo than in those who received abatacept (53% vs 20%, P = 0.0003). There was no significant difference in adverse events between the two groups. The authors noted that clinical improvements during the open-label treatment were maintained in many patients randomly assigned to receive placebo.

These results suggest that abatacept therapy is a suitable alternative for children with JIA who fail to respond or have intolerance to DMARDs.