Neonatal-Onset Multisystem Inflammatory Disease Responsive to Interleukin-1β Inhibition

Among patients with neonatal-onset multisystem inflammatory disease — a disorder characterized by fever, urticarial rash, aseptic meningitis, deforming arthropathy, hearing loss, and mental retardation — treatment with the interleukin-1 antagonist anakinra markedly improved clinical and radiologic manifestations of disease (including central nervous system manifestations) and decreased serum markers of inflammation. Anakinra withdrawal resulted in a flare of disease within days; retreatment led to rapid improvement. Among patients with neonatal-onset multisystem inflammatory disease, treatment with the interleukin-1 antagonist anakinra markedly improved clinical and radiologic manifestations of disease and decreased serum markers of inflammation. Neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic cutaneous articular (CINCA) syndrome, is a rare chronic inflammatory disease. 1 , 2 An urticaria-like rash develops within the first six weeks of life, and a characteristic bony overgrowth predominantly involving the knees develops in most affected children. Central nervous system (CNS) manifestations include chronic aseptic meningitis, increased intracranial pressure, cerebral atrophy, ventriculomegaly, and chronic papilledema, with associated optic-nerve atrophy and loss of vision, mental retardation, seizures, and sensorineural hearing loss. Other manifestations include short stature, hepatosplenomegaly, leukocytosis, and an elevation in serum levels of amyloid A and C-reactive protein and . . .