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ArchivistTreatment for cryopyrin-associated periodic syndrome
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The term cryopyrin-associated periodic syndrome (CAPS) embraces several rare, inherited, inflammatory disorders including the familial cold autoinflammatory syndrome, the Muckle-Wells syndrome and neonatal-onset multisystem inflammatory disorder (otherwise known as the chronic infantile neurologic, cutaneous and articular syndrome). Common features include onset in infancy, severe fatigue, fever, myalgia, anaemia, and inflammation of skin, eyes, bones, joints and meninges. Sensorineural deafness and intellectual impairment may occur and systemic AA amyloidosis develops in about a quarter of all patients. CAPS is caused by mutations in the cryopyrin gene, NLRP3, causing overproduction of interleukin-1β. Now (Helen J Lachmann and colleagues. New England Journal of Medicine 2009;360:2416–25; see also Editorial, ibid: 2467–70) a human anti-interleukin-1β monoclonal antibody (canakinumab) has been shown to be a highly effective treatment for CAPS. Thirty four of 35 patients (four aged 4–16 years) responded completely to a single dose of canakinumab. Of the responders, 31
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