Anakinra for cryopyrin-associated periodic syndrome. (January 2014)

• Record Type: Journal Article
• Authors: Koné-Paut, Isabelle; Galeotti, Caroline
• Is Part Of: Expert review of clinical immunology. Volume 10:Number 1(2014)
• Page Start: 7
• Page End: 18
• Publication Date: 2014-01
• Subjects: Clinical immunology -- Periodicals; 616.079
• DOI: 10.1586/1744666X.2014.861325
• Languages: English
• ISSNs: 1744-666X

Abstract

<abstract> <title> <x xml:space="preserve">Abstract</x> </title> <p>Cryopyrin-associated periodic syndrome (CAPS) is a very rare auto-inflammatory syndrome, which has recently served as a pure model of IL-1β-driven diseases. CAPS is caused by mutations into the <italic>NLRP3</italic> gene that encodes crypoyrin, which serves as a receptor of the innate immunity that senses danger signals and pathogens. Constitutive activation of cryopyrin in CAPS leads to an excessive secretion of IL-1β. CAPS patients experience symptoms of systemic inflammation, intense fatigue and have poor quality of life. In the most severe forms, they may develop serious organ damage such as visual and hearing impairment, neurological deterioration and renal insufficiency. Anti-IL-1 drugs are effective in treating symptoms of almost all CAPS patients and have radically transformed their lives. We describe the history of the 'revival' of CAPS patients through anti-IL-1 treatments with a special focus on anakinra, the first drug used in cohorts with variable disease severity and number of patients.</p> </abstract>