@article {1670, title = {Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with Anakinra.}, journal = {J Pediatr}, volume = {157}, year = {2010}, month = {2010 Aug}, pages = {310-315.e1}, 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).

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.

}, keywords = {Adolescent, Adult, Case-Control Studies, Child, Child, Preschool, Cryopyrin-Associated Periodic Syndromes, Female, Follow-Up Studies, Humans, Inflammation, Interleukin 1 Receptor Antagonist Protein, Interleukin-1, Male, Phenotype, Quality of Health Care, Quality of Life, Questionnaires, Syndrome, Treatment Outcome}, issn = {1097-6833}, doi = {10.1016/j.jpeds.2010.02.040}, author = {Lepore, Loredana and Paloni, Giulia and Caorsi, Roberta and Alessio, Maria and Rigante, Donato and Ruperto, Nicola and Cattalini, Marco and Tommasini, Alberto and Zulian, Francesco and Ventura, Alessando and Martini, Alberto and Gattorno, Marco} }