Follow-up and quality of life of patients with cryopyrin-associated periodic syndromes treated with Anakinra.
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.
Lepore
Loredana
L
IRCCS Burlo Garofolo, the Department of Reproductive and Developmental Sciences, University of Trieste, Italy.
Paloni
Giulia
G
Caorsi
Roberta
R
Alessio
Maria
M
Rigante
Donato
D
Ruperto
Nicola
N
Cattalini
Marco
M
Tommasini
Alberto
A
Zulian
Francesco
F
Ventura
Alessando
A
Martini
Alberto
A
Gattorno
Marco
M
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
D053590:Interleukin 1 Receptor Antagonist Protein; D007375:Interleukin-1