Follow-Up and Quality of Life of Patients with Cryopyrin-Associated Periodic Syndromes Treated with Anakinra

Lepore, Loredana; Paloni, Giulia; Caorsi, Roberta; Alessio, Maria; Rigante, Donato; Ruperto, Nicola; Cattalini, Marco; Tommasini, Alberto; Zulian, Francesco; Ventura, Alessando; Martini, Alberto; Gattorno, Marco

doi:10.1016/j.jpeds.2010.02.040

« Previous Next »The Journal of Pediatrics
Volume 157, Issue 2 , Pages 310-315.e1, August 2010

Follow-Up and Quality of Life of Patients with Cryopyrin-Associated Periodic Syndromes Treated with Anakinra

Loredana Lepore, MD
- IRCCS Burlo Garofolo, the Department of Reproductive and Developmental Sciences, University of Trieste, Italy
Giulia Paloni, MD
- IRCCS Burlo Garofolo, the Department of Reproductive and Developmental Sciences, University of Trieste, Italy
Roberta Caorsi, MD
- II Division of Pediatrics, “G. Gaslini” Institute for Children and Department of Pediatrics, Genoa, Italy
Maria Alessio, MD
- Department of Pediatrics, Federico II Hospital, Naples, Italy
Donato Rigante, MD
- Department of Pediatric Sciences, Catholic University of Sacred Heart, Rome
Nicola Ruperto, MD
- II Division of Pediatrics, “G. Gaslini” Institute for Children and Department of Pediatrics, Genoa, Italy
Marco Cattalini, MD
- Department of Rheumatology and Immunology, Brescia Hospital and University of Brescia, Italy
Alberto Tommasini, MD
- IRCCS Burlo Garofolo, the Department of Reproductive and Developmental Sciences, University of Trieste, Italy
Francesco Zulian, MD
- Pediatric Rheumatology Unit, Department of Pediatrics, University of Padova, Italy
Alessando Ventura, MD
- IRCCS Burlo Garofolo, the Department of Reproductive and Developmental Sciences, University of Trieste, Italy
Alberto Martini, MD
- II Division of Pediatrics, “G. Gaslini” Institute for Children and Department of Pediatrics, Genoa, Italy
Marco Gattorno, MD
- II Division of Pediatrics, “G. Gaslini” Institute for Children and Department of Pediatrics, Genoa, Italy
- Reprint requests: Marco Gattorno, MD, UC Pediatria II, G. Gaslini Institute, Largo G. Gaslini 5, 16147 Genova, Italy.

Received 15 October 2009; received in revised form 6 January 2010; accepted 19 February 2010. published online 17 May 2010.

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.

CAPS, Cryopyrin associated periodic syndromes, CHQ-PF50, Child Health Questionaire, CINCA, Chronic infantile neurological cutaneous and auricular syndrome, IL, Interleukin, HRQoL, Health-Related Quality of Life, PhS, Physical summary score, PsS, Psychosocial summary score, MWS, Muckle-Wells syndrome