title: Anakinra for cryopyrin-associated periodic syndrome.
abstract: 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 NLRP3 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.
authors.affiliation: Division of Paediatric Rheumatology, Reference Centre for Autoinflammatory Disorders CEREMAI, Bicêtre Hospital, University of Paris SUD, Le Kremlin Bicêtre Cedex, France.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Koné-Paut
authors.forename: Caroline
authors.initials: C
authors.lastname: Galeotti
mesh_terms: D018501:Antirheumatic Agents / Q000627:therapeutic use*; D002352:Carrier Proteins / Q000097:blood / Q000276:immunology; D056587:Cryopyrin-Associated Periodic Syndromes / Q000097:blood / Q000188:drug therapy* / Q000276:immunology; D006801:Humans; D053590:Interleukin 1 Receptor Antagonist Protein / Q000627:therapeutic use*; D053583:Interleukin-1beta / Q000037:antagonists & inhibitors* / Q000097:blood / Q000276:immunology; D008954:Models, Biological; D000071199:NLR Family, Pyrin Domain-Containing 3 Protein; D012720:Severity of Illness Index
chemical_list: D018501:Antirheumatic Agents; D002352:Carrier Proteins; C508585:IL1B protein, human; D053590:Interleukin 1 Receptor Antagonist Protein; D053583:Interleukin-1beta; D000071199:NLR Family, Pyrin Domain-Containing 3 Protein; C453881:NLRP3 protein, human