title: Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with Anakinra.
abstract: OBJECTIVE
  To evaluate the quality of life and long-term follow-up of patients enrolled in the Italian registry of cryopyrin-associated periodic syndromes (CAPS).
  
  
  METHODS
  Since 2004, 20 patients with CAPS were enrolled in a common registry from different Italian Centers of Pediatric Rheumatology; 14 patients were treated with Anakinra in an open fashion. Both treated and untreated patients were routinely followed according to standard of care. The Child Health Questionnaire (CHQ-PF 50) was used to assess the health-related quality of life.
  
  
  RESULTS
  The mean duration of follow-up was 37.5 months. In all treated patients, a complete and persistent control of the inflammatory manifestations was observed with no further progression of the disease. At enrollment in the registry, patients showed a poorer health-related quality of life than healthy children in both physical and the psychosocial summary scores. Treatment was associated with a dramatic and sustained amelioration of a variety of measures of poor quality of life, particularly in those concerning the global health perception, bodily pain-discomfort, and other physical domains.
  
  
  CONCLUSIONS
  Long-term IL-1 blockade produces a significant and persistent improvement in the clinical manifestations associated with the disease and on the overall quality of life.
authors.affiliation: IRCCS Burlo Garofolo, the Department of Reproductive and Developmental Sciences, University of Trieste, Italy.
authors.forename: Loredana
authors.initials: L
authors.lastname: Lepore
authors.forename: Giulia
authors.initials: G
authors.lastname: Paloni
authors.forename: Roberta
authors.initials: R
authors.lastname: Caorsi
authors.forename: Maria
authors.initials: M
authors.lastname: Alessio
authors.forename: Donato
authors.initials: D
authors.lastname: Rigante
authors.forename: Nicola
authors.initials: N
authors.lastname: Ruperto
authors.forename: Marco
authors.initials: M
authors.lastname: Cattalini
authors.forename: Alberto
authors.initials: A
authors.lastname: Tommasini
authors.forename: Francesco
authors.initials: F
authors.lastname: Zulian
authors.forename: Alessando
authors.initials: A
authors.lastname: Ventura
authors.forename: Alberto
authors.initials: A
authors.lastname: Martini
authors.forename: Marco
authors.initials: M
authors.lastname: Gattorno
mesh_terms: D000293:Adolescent; D000328:Adult; D016022:Case-Control Studies; D002648:Child; D002675:Child, Preschool; D056587:Cryopyrin-Associated Periodic Syndromes / Q000188:drug therapy* / Q000503:physiopathology*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007249:Inflammation; D053590:Interleukin 1 Receptor Antagonist Protein / Q000627:therapeutic use*; D007375:Interleukin-1 / Q000037:antagonists & inhibitors; D008297:Male; D010641:Phenotype; D011787:Quality of Health Care; D011788:Quality of Life; D011795:Surveys and Questionnaires; D013577:Syndrome; D016896:Treatment Outcome
chemical_list: D053590:Interleukin 1 Receptor Antagonist Protein; D007375:Interleukin-1