Efficacy and safety of canakinumab therapy in paediatric patients with cryopyrin-associated periodic syndrome: a single-centre, real-world experience

• Published in: Rheumatology (Oxford, England) England), 2014-04, Vol.53 (4), p.665-670
• Main Authors: Russo, Ricardo A G, Melo-Gomes, Sonia, Lachmann, Helen J, Wynne, Karen, Rajput, Kaukab, Eleftheriou, Despina, Edelsten, Clive, Hawkins, Philip N, Brogan, Paul A
• Format: Article
• Language: English
• Subjects: Adolescent; Antibodies, Monoclonal - therapeutic use; Blood Sedimentation; C-Reactive Protein - immunology; Child; Child, Preschool; Cryopyrin-Associated Periodic Syndromes - drug therapy; Cryopyrin-Associated Periodic Syndromes - immunology; Female; Hemoglobins - immunology; Humans; Interleukin-1beta - antagonists & inhibitors; Male; Serum Amyloid A Protein - immunology; Severity of Illness Index; Treatment Outcome
• ISSN: 1462-0324; 1462-0332
• DOI: 10.1093/rheumatology/ket415

The aim of this study was to determine the short- and long-term efficacy and safety of 8-weekly canakinumab therapy in children with cryopyrin-associated periodic syndromes (CAPS) in routine clinical practice. A single-centre observational study was performed. Patients were assessed every 8 weeks at a dedicated clinic. Standardized assessments were the 10-domains DAS for CAPS, acute phase reactants (APRs), physician's global assessment of disease activity, Child Health Assessment Questionnaire (CHAQ) and Child Health Questionnaire Parent Form 28 (CHQPF-28). The primary endpoint was clinical improvement, defined as a reduction of DAS score 8 weeks after commencing therapy. Secondary endpoints included sustained clinical improvement in APRs, relapses, CHAQ score and CHQPF-28 score. Ten children with CAPS [eight Muckle-Wells syndrome (MWS), two chronic infantile cutaneous neurological articular (CINCA); median age 6.3 years] received 8-weekly canakinumab treatments at 2-8.7 mg/kg for a median of 21 months (range 12-31 months). Nine of 10 patients improved after the first dose: baseline median DAS of 7.5/20 decreased to 3.5/20 at 8 weeks (P = 0.04). This clinical improvement was sustained at a median follow-up of 21 months (range 12-31 months). Children with CINCA required higher doses of canakinumab than those with MWS. CHAQ and CHQ scores indicated improvement in functioning and health-related quality of life (HRQoL). Treatment was well tolerated, with no injection site reactions and no serious infections. Canakinumab, although costly, is a safe and effective treatment for CAPS in children, leading to sustained improvement in disease activity, serological markers, functional ability and HRQoL.