TY - JOUR T1 - Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with Anakinra. JF - J Pediatr Y1 - 2010 A1 - Lepore, Loredana A1 - Paloni, Giulia A1 - Caorsi, Roberta A1 - Alessio, Maria A1 - Rigante, Donato A1 - Ruperto, Nicola A1 - Cattalini, Marco A1 - Tommasini, Alberto A1 - Zulian, Francesco A1 - Ventura, Alessando A1 - Martini, Alberto A1 - Gattorno, Marco KW - Adolescent KW - Adult KW - Case-Control Studies KW - Child KW - Child, Preschool KW - Cryopyrin-Associated Periodic Syndromes KW - Female KW - Follow-Up Studies KW - Humans KW - Inflammation KW - Interleukin 1 Receptor Antagonist Protein KW - Interleukin-1 KW - Male KW - Phenotype KW - Quality of Health Care KW - Quality of Life KW - Questionnaires KW - Syndrome KW - Treatment Outcome AB -

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).

STUDY DESIGN: 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.

VL - 157 IS - 2 U1 - http://www.ncbi.nlm.nih.gov/pubmed/20472245?dopt=Abstract ER -