Randomized Trial of Tocilizumab in Systemic Juvenile Idiopathic Arthritis

In a randomized trial involving children with severe systemic juvenile idiopathic arthritis, the anti–interleukin-6 receptor antibody tocilizumab was effective (response rate, 85% with tocilizumab vs. 24% with placebo). Adverse events included serious infections and neutropenia. Systemic juvenile idiopathic arthritis (JIA) is characterized by chronic arthritis, systemic manifestations (spiking fever, rash, hepatosplenomegaly, lymphadenopathy, and serositis), and substantially elevated inflammatory markers. 1 It is the most severe subtype of JIA; approximately half the patients have an unremitting course of chronic polyarthritis (with or without persistent systemic features). Substantial joint damage and disability often develop in these patients. 2 , 3 Treatment remains challenging because of the limited efficacy of methotrexate 4 and tumor necrosis factor inhibitors 5 , 6 and because of the major toxicity of high-dose glucocorticoids. Efficacy of the interleukin-1 inhibitor anakinra has been reported in a subset of patients. 7 – . . .