Efficacy and safety of canakinumab in cryopyrin-associated periodic syndromes: results from a Spanish cohort.

Autor:	Anton J; Paediatric Rheumatology Unit, Hospital Sant Joan de Déu, Universitat de Barcelona, Esplugues, Spain. janton@hsjdbcn.org., Calvo I; Paediatric Rheumatology Unit, Hospital Universitario y Politecnico La Fe, Valencia, Spain., Fernández-Martin J; Department of Internal Medicine, Hospital Meixoeiro, Vigo, Spain., Gamir ML; Department of Paediatric Rheumatology, Hospital Ramón y Cajal, Madrid, Spain., Merino R; Department of Paediatric Rheumatology. Hospital La Paz, Madrid, Spain., Jimenez-Treviño S; Department of Paediatrics, Hospital Universitario Central de Asturias, Oviedo, Spain., Sevilla B; Department of Paediatrics, Hospital Universitario San Cecilio, Granada, Spain., Cabades F; Department of Internal Medicine, Hospital de Vinaros, Vinaros, Spain., Bou R; Paediatric Rheumatology Unit, Hospital Sant Joan de Déu, Universitat de Barcelona, Esplugues, and Department of Paediatrics, Corporacio Sanitaria Parc Taulí, Sabadell, Spain., Arostegui JI; Department of Immunology-CDB, Hospital Clínic-IDIBAPS, Barcelona, Spain.
Jazyk:	angličtina
Zdroj:	Clinical and experimental rheumatology [Clin Exp Rheumatol] 2015 Nov-Dec; Vol. 33 (6 Suppl 94), pp. S67-71. Date of Electronic Publication: 2015 Aug 05.
Abstrakt:	Objectives: Cryopyrin-associated periodic syndromes (CAPS) are dominantly-inherited autoinflammatory diseases. The uncontrolled IL-1β overproduction observed in these patients is the rational basis to treat them with anti-IL-1 drugs. The objective of this study was to evaluate the efficacy and safety of treatment with the long-lasting fully humanised anti-IL-1β monoclonal antibody canakinumab in a Spanish cohort of patients with CAPS. Methods: Clinical and laboratory data of CAPS patients carrying a heterozygous germline NLRP3 mutation were obtained. The initial treatment scheme with canakinumab was 150 mg/8 weeks administered subcutaneously in adult patients and 2 mg/kg/8 weeks in paediatric patients. Results: Eight unrelated patients were enrolled. Canakinumab was the first anti-IL-1 drug used in three of them; five were already receiving anakinra. The clinical response to the initial canakinumab scheme was positive in all patients, and was quickly observed in the first 24-72 hours. Four required increasing the frequency and/or dose of canakinumab. A limited or no efficacy in those symptoms related to consequence of the deforming arthropathy and neurosensorial deafness was observed. The adverse side effects were restricted to infectious complications in a small percentage of patients. The treatment was well tolerated by all patients, with no reactions at drug site injections. Conclusions: Canakinumab caused fast and sustained remissions in most clinical and biochemical manifestations in all enrolled patients, with a limited efficacy in the structural lesions. Dose adjustments seem to be necessary for children and/or for patients with the most severe CAPS phenotypes. Treatment was well tolerated with a low incidence of adverse effects.
Databáze:	MEDLINE
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