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Canakinumab treat-to target strategies increase complete response rate in CAPS
© Kuemmerle-Deschner et al. 2015
Published: 28 September 2015
Cryopyrin-associated periodic syndrome (CAPS) is a heterogeneous group of diseases characterized by excessive Interleukin-1β (IL-1 β) release resulting in severe systemic and organ inflammation. Canakinumab targets IL-1β and is approved at standard dose for children and adults with all CAPS phenotypes. Limited data are available regarding real-life effectiveness of canakinumab in patients living with CAPS. Therefore the aim of the study was to evaluate the real-life dosing practice and effectiveness of canakinumab in CAPS.
A multi-center study of consecutive children and adults with CAPS treated with canakinumab was performed. Demographics, CAPS phenotype and disease activity, inflammatory markers and canakinumab treatment strategy were recorded. Treatment response was assessed using CAPS disease activity scores, CRP and/or SAA levels. Comparisons between age groups, CAPS phenotypes and centers were conducted.
A total of 68 CAPS patients at nine centers were included, these were 31 males and 37 females; median age was 25 years and 27 (40%) were children. All CAPS phenotypes were represented. Median follow up was 28 months. Overall, complete response (CR) was seen in 72% of CAPS patients, significantly less often in severe (14%) than in mild CAPS phenotypes (79%). Only 53% attained CR on standard dose. Dose increase was more commonly required in children (56%) than in adults (22%). Centers with treat-to-target approach achieved significantly higher CR rates (94% vs 50%).
Real-life effectiveness of canakinumab in CAPS was significantly lower than in controlled trials. Treat-to-target strategies may improve the outcome of children and adults living with CAPS.
This article is published under license to BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.