Real-life effectiveness of canakinumab in cryopyrin-associated periodic syndrome
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Real-life effectiveness of canakinumab in cryopyrin-associated periodic syndrome. / Kuemmerle-Deschner, Jasmin B; Hofer, Ferdinand; Endres, Theresa; Kortus-Goetze, Birgit; Blank, Norbert; Weißbarth-Riedel, Elisabeth; Schuetz, Catharina; Kallinich, Tilmann; Krause, Karoline; Rietschel, Christoph; Horneff, Gerd; Benseler, Susanne M.
in: RHEUMATOLOGY, Jahrgang 55, Nr. 4, 04.2016, S. 689-96.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Real-life effectiveness of canakinumab in cryopyrin-associated periodic syndrome
AU - Kuemmerle-Deschner, Jasmin B
AU - Hofer, Ferdinand
AU - Endres, Theresa
AU - Kortus-Goetze, Birgit
AU - Blank, Norbert
AU - Weißbarth-Riedel, Elisabeth
AU - Schuetz, Catharina
AU - Kallinich, Tilmann
AU - Krause, Karoline
AU - Rietschel, Christoph
AU - Horneff, Gerd
AU - Benseler, Susanne M
N1 - © The Author 2015. Published by Oxford University Press on behalf of the British Society for Rheumatology. All rights reserved. For Permissions, please email: journals.permissions@oup.com.
PY - 2016/4
Y1 - 2016/4
N2 - OBJECTIVE: Cryopyrin-associated periodic syndrome (CAPS) is a heterogeneous group of diseases characterized by excessive IL-1β 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 for the real-life effectiveness of canakinumab in patients living with CAPS. Therefore the aim of the study was to evaluate the real-life dosing and effectiveness of canakinumab in CAPS.METHODS: A multi-centre 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 serum amyloid A levels. Comparisons between age groups, CAPS phenotypes and centres were conducted.RESULTS: A total of 68 CAPS patients at nine centres were included. All CAPS phenotypes were represented. Thirty-seven (54%) patients were females, the median age was 25 years and 27 (40%) were children, and the 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 canakinumab. Dose increase was more commonly required in children (56%) than in adults (22%). Centres with a treat-to-target approach had significantly higher CR rates (94 vs 50%).CONCLUSION: 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.
AB - OBJECTIVE: Cryopyrin-associated periodic syndrome (CAPS) is a heterogeneous group of diseases characterized by excessive IL-1β 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 for the real-life effectiveness of canakinumab in patients living with CAPS. Therefore the aim of the study was to evaluate the real-life dosing and effectiveness of canakinumab in CAPS.METHODS: A multi-centre 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 serum amyloid A levels. Comparisons between age groups, CAPS phenotypes and centres were conducted.RESULTS: A total of 68 CAPS patients at nine centres were included. All CAPS phenotypes were represented. Thirty-seven (54%) patients were females, the median age was 25 years and 27 (40%) were children, and the 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 canakinumab. Dose increase was more commonly required in children (56%) than in adults (22%). Centres with a treat-to-target approach had significantly higher CR rates (94 vs 50%).CONCLUSION: 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.
KW - Adolescent
KW - Adult
KW - Age Factors
KW - Aged
KW - Antibodies, Monoclonal
KW - Child
KW - Child, Preschool
KW - Cryopyrin-Associated Periodic Syndromes
KW - Dose-Response Relationship, Drug
KW - Female
KW - Humans
KW - Interleukin-1beta
KW - Male
KW - Middle Aged
KW - Treatment Outcome
KW - Young Adult
U2 - 10.1093/rheumatology/kev416
DO - 10.1093/rheumatology/kev416
M3 - SCORING: Journal article
C2 - 26667214
VL - 55
SP - 689
EP - 696
JO - RHEUMATOLOGY
JF - RHEUMATOLOGY
SN - 1462-0324
IS - 4
ER -
