title: Interleukin-1 targeting drugs in familial Mediterranean fever: a case series and a review of the literature.
abstract: OBJECTIVE
  Familial Mediterranean fever (FMF) is an autosomal-recessive autoinflammatory disorder common in Mediterranean populations. FMF is associated with mutations of the MEFV gene, which encodes pyrin. Functional studies suggest that pyrin is implicated in the maturation and secretion of IL-1. The IL-1 receptor antagonist or anti-IL1 monoclonal antibody may therefore represent a new approach to treat FMF. The aim of this report was to evaluate and discuss treatment of FMF with interleukin-1 targeting drugs.
  
  
  METHODS
  Electronic mailing lists of French pediatric and adult rheumatologist associations were used to call for FMF patients treated with interleukin-1 antagonists. A search for published FMF patients treated with interleukin-1 targeting drugs was performed by screening PubMed.
  
  
  RESULTS
  Here, we report 7 cases of FMF patients treated with anakinra and/or canakinumab and discuss the clinical situations that may indicate the use of IL-1 blocking agents in FMF. The use of interleukin-1 targeting drugs was beneficial to all patients. The reasons for using interleukin-1 targeting drugs in FMF patients were as follows: (1) incomplete control of disease activity despite colchicine treatment; (2) high serum amyloid A levels despite colchicine treatment; (3) impossibility to use colchicine treatment because of severe side effects; (4) FMF in association with vasculitis.
  
  
  CONCLUSIONS
  Interleukin-1 targeting drugs may be good candidates when looking for an alternative or supplementary treatment to colchicine. These observations highlight the need for controlled trials to further evaluate the safety and efficacy of interleukin-1 antagonists in FMF patients.
authors.affiliation: Division of Paediatric Rheumatology, CEREMAI, Hôpital de Bicêtre, University of Paris Sud, Le Kremlin Bicêtre Cedex, France. ulrich.meinzer@bct.aphp.fr
authors.forename: Ulrich
authors.initials: U
authors.lastname: Meinzer
authors.forename: Pierre
authors.initials: P
authors.lastname: Quartier
authors.forename: Jean-François
authors.initials: JF
authors.lastname: Alexandra
authors.forename: Véronique
authors.initials: V
authors.lastname: Hentgen
authors.forename: Frédérique
authors.initials: F
authors.lastname: Retornaz
authors.forename: Isabelle
authors.initials: I
authors.lastname: Koné-Paut
mesh_terms: D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D002648:Child; D010505:Familial Mediterranean Fever / Q000188:drug therapy*; D005260:Female; D006801:Humans; D053590:Interleukin 1 Receptor Antagonist Protein / Q000627:therapeutic use*; D053583:Interleukin-1beta / Q000037:antagonists & inhibitors*; D008297:Male; D008875:Middle Aged; D016896:Treatment Outcome
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D053590:Interleukin 1 Receptor Antagonist Protein; D053583:Interleukin-1beta; C541220:canakinumab