Results from a 2006 trial of a biologic found it to be useful in treating a rare form of juvenile arthritis. Neonatal-onset multisystem inflammatory disease is a rare chronic inflammatory disease, characterized by rash, deforming arthritis, meningitis, vision loss, mental retardation and hearing loss. It affects many aspects of a person’s life and is one of a family of diseases called hereditary systemic autoinflammatory disorders. Results of a 2006 trial of anakinra (Kineret) found the drug significantly decreased disease symptoms and organ damage in both children and adults with the disease. The trial revealed that rash, measures of inflammation, intracranial pressure and hearing all improved with treatment.

Mutations in a gene that encodes cryopyrin, a protein involved in inflammation, are responsible for about 60 percent of cases of the disease. Therapies aim to reduce inflammation and, to date, have included high-dose corticosteroids, DMARDs and a subset of biologics called tumor necrosis factor inhibitors. Although these medications are moderately effective, inflammation has persisted in most children. A large multicenter team of scientists found that the interleukin-1–receptor antagonist anakinra was effective in treating the disease, giving people an additional, perhaps more effective, treatment option (New England Journal of Medicine August 10, 2006).