title: Biologic drugs in autoinflammatory syndromes.
abstract: OBJECTIVE
  Inherited autoinflammatory syndromes are conditions caused by mutations of proteins playing a pivotal role in the regulation of the innate immunity leading to an uncontrolled inflammation. The understanding of the molecular pathways involved in these disorders has shed a new light on the pattern of activation and maintenance of the inflammatory response and disclosed new molecular therapeutic targets. In this review we give a start of the art of the use of biologics in these disorders.
  
  
  OBJECTIVE
  The dramatic response to anti IL-1 drugs in cryopyrin-associated periodic syndromes represents the brightest example of the possibility to completely dampen inflammation in these severe disorders with the selective blockade of a single pivotal cytokine. Periodic fevers are characterized by recurrent episodes of fever, usually treated with on demand steroids. However the increasing frequency of fever episodes or the development of a chronic disease course may require a continuous long-term treatment, with anti-TNF or IL-1 blockers in mevalonate kinase deficiency and TNF-receptor associated periodic syndrome. Anti-IL-1 treatment is also effective in FMF patients resistant or partially responsive to colchicine. The deficiency of the interleukin-1-receptor antagonist (DIRA) is caused by mutations in the gene encoding for the interleukin-1 receptor antagonist (IL-1Ra). In this case t he recombinant IL-1Ra (anakinra) is the treatment of choice. Due to their extreme rarity the response to the available biologic drugs in other autoinflammatory diseases is still largely anecdotal.
authors.affiliation: Department of Paediatrics, University of Genoa, Pediatria II, Reumatologia, IRCCS G. Gaslini, Genoa, Italy.
authors.forename: Roberta
authors.initials: R
authors.lastname: Caorsi
authors.forename: Silvia
authors.initials: S
authors.lastname: Federici
authors.forename: Marco
authors.initials: M
authors.lastname: Gattorno
mesh_terms: D001685:Biological Factors / Q000627:therapeutic use*; D056660:Hereditary Autoinflammatory Diseases / Q000188:drug therapy*; D006801:Humans; D053583:Interleukin-1beta / Q000037:antagonists & inhibitors
chemical_list: D001685:Biological Factors; D053583:Interleukin-1beta