title: Interleukin-1 targeting treatment in familial Mediterranean fever: an experience of pediatric patients.
abstract: OBJECTIVE
  The aim of this report was to evaluate and discuss treatment of pediatric familial Mediterranean fever (FMF) patients with anti-interleukin1 (IL-1) agents.
  
  
  METHODS
  Refractory or colchicine unresponsive FMF was described as severe and frequent attacks and/or having high acute phase reactance levels despite having a maximum dose of colchicine (2 mg/day). Disease course, adverse effects, duration of follow-up, treatment protocols, responses to the therapies were discussed.
  
  
  RESULTS
  Eight patients (6 male, 2 female) having refractory FMF were identified. Mediterranean fever (MEFV) gene analyses revealed homozygous M694V mutations in six patients and heterozygote M694V mutations in one patient and no mutation in one patient. They were all treated with anakinra and/or canakinumab. The use of anti-IL-1 drugs was beneficial to all patients. None of them had any severe adverse effects due to the therapy.
  
  
  CONCLUSIONS
  Anakinra and canakinumab were effective in patient refractory to colchicine treatment as shown both in our series and in the literature. Therefore, controlled trials are needed to evaluate the safety and long-term efficacy of IL-1 targeting agents in colchicine resistant patients.
authors.affiliation: Department of Pediatric Rheumatology, Ankara, Child Health, Hematology and Oncology Education and Research Hospital , Ankara , Turkey.
authors.forename: Özge
authors.initials: Ö
authors.lastname: Başaran
authors.forename: Nermin
authors.initials: N
authors.lastname: Uncu
authors.forename: Banu Acar
authors.initials: BA
authors.lastname: Çelikel
authors.forename: Aysel
authors.initials: A
authors.lastname: Taktak
authors.forename: Gökçe
authors.initials: G
authors.lastname: Gür
authors.forename: Nilgun
authors.initials: N
authors.lastname: Cakar
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002985:Clinical Protocols; D003078:Colchicine / Q000627:therapeutic use*; D010505:Familial Mediterranean Fever / Q000097:blood / Q000188:drug therapy* / Q000235:genetics; D005260:Female; D005820:Genetic Testing; D006074:Gout Suppressants / Q000627:therapeutic use; D006801:Humans; D007375:Interleukin-1 / Q000235:genetics* / Q000378:metabolism; D008297:Male; D009154:Mutation
chemical_list: D006074:Gout Suppressants; D007375:Interleukin-1; D003078:Colchicine
keywords: Anti IL-1 treatment; FMF; Pediatric