title: Persistent efficacy of anakinra in patients with tumor necrosis factor receptor-associated periodic syndrome.
abstract: OBJECTIVE
  To evaluate the efficacy and safety of treatment with the interleukin-1 receptor antagonist anakinra in patients with tumor necrosis factor receptor-associated periodic syndrome (TRAPS) requiring high cumulative doses of steroids.
  
  
  METHODS
  Four children (mean age 9.1 years [range 4-13 years]) and 1 adult (age 33 years) with TRAPS were enrolled in the study. The 3 children with cysteine mutations (C52Y, C55Y, C43R) had prolonged and frequent attacks of fever. One child with the R92Q mutation and the adult patient with the C43R mutation displayed a more chronic disease course, with fluctuating, nearly continuous symptoms and persistent elevation of acute-phase reactant levels (including serum amyloid A [SAA]). All patients were treated with anakinra (1.5 mg/kg/day).
  
  
  RESULTS
  All of the patients had a prompt response to anakinra, with disappearance of symptoms and normalization of acute-phase reactant levels, including SAA. In all pediatric patients, anakinra was withdrawn after 15 days of treatment. After a few days (mean 5.6 days [range 3-8]) a disease relapse occurred, which dramatically responded to reintroduction of anakinra. During the following period of observation (mean 11.4 months [range 4-20 months]), the patients did not experience episodes of fever or other disease-related clinical manifestations. Levels of acute-phase reactants remained in the normal range. No major adverse reactions or severe infections were observed.
  
  
  CONCLUSIONS
  Continuous treatment with anakinra effectively controlled both the clinical and laboratory manifestations in patients with TRAPS and prevented disease relapses.
authors.affiliation: Istituto Giannina Gaslini, Genoa, Italy. marcogattorno@ospedale-gaslini.ge.it
authors.forename: M
authors.initials: M
authors.lastname: Gattorno
authors.forename: M A
authors.initials: MA
authors.lastname: Pelagatti
authors.forename: A
authors.initials: A
authors.lastname: Meini
authors.forename: L
authors.initials: L
authors.lastname: Obici
authors.forename: R
authors.initials: R
authors.lastname: Barcellona
authors.forename: S
authors.initials: S
authors.lastname: Federici
authors.forename: A
authors.initials: A
authors.lastname: Buoncompagni
authors.forename: A
authors.initials: A
authors.lastname: Plebani
authors.forename: G
authors.initials: G
authors.lastname: Merlini
authors.forename: A
authors.initials: A
authors.lastname: Martini
mesh_terms: D000293:Adolescent; D000328:Adult; D018501:Antirheumatic Agents / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D010505:Familial Mediterranean Fever / Q000188:drug therapy*; D005260:Female; D006801:Humans; D053590:Interleukin 1 Receptor Antagonist Protein / Q000627:therapeutic use*; D008297:Male; D018124:Receptors, Tumor Necrosis Factor
chemical_list: D018501:Antirheumatic Agents; D053590:Interleukin 1 Receptor Antagonist Protein; D018124:Receptors, Tumor Necrosis Factor