Jasmin B Kuemmerle-Deschner1, Ferdinand Hofer2, Theresa Endres2, Birgit Kortus-Goetze3, Norbert Blank4, Elisabeth Weißbarth-Riedel5, Catharina Schuetz6, Tilmann Kallinich7, Karoline Krause8, Christoph Rietschel9, Gerd Horneff10, Susanne M Benseler11. 1. Division of Pediatric Rheumatology, Department of Pediatrics, University Children's Hospital Tuebingen, kuemmerle.deschner@uni-tuebingen.de. 2. Division of Pediatric Rheumatology, Department of Pediatrics, University Children's Hospital Tuebingen. 3. Division of Nephrology, University of Marburg. 4. Haematology, Oncology and Rheumatology, University Hospital Heidelberg. 5. Pediatric Rheumatology Clinics, University Hospital Eppendorf, Hamburg. 6. Department of Pediatrics and Adolescents Medicine, University Hospital Ulm. 7. Childrens Hospital, Section Rheumatology, Charite Campus Virchow, Berlin. 8. Allergie-Centrum Charite, Department for Dermatology, Charite Campus Mitte, Berlin. 9. Department of Rheumatology, Clementine Childrens Hospital, Frankfurt. 10. Centre for Pediatric Rheumatology Sankt Augustin, Asklepios Klinik Sankt Augustin, Sankt Augustin, Germany. 11. Division of Pediatric Rheumatology, Department of Pediatrics, University Children's Hospital Tuebingen, and Rheumatology, Alberta Children's Hospital, University of Calgary, Alberta, Canada.
Abstract
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.
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.
Authors: Micol Romano; Z Serap Arici; David Piskin; Sara Alehashemi; Daniel Aletaha; Karyl Barron; Susanne Benseler; Roberta A Berard; Lori Broderick; Fatma Dedeoglu; Michelle Diebold; Karen Durrant; Polly Ferguson; Dirk Foell; Jonathan S Hausmann; Olcay Y Jones; Daniel Kastner; Helen J Lachmann; Ronald M Laxer; Dorelia Rivera; Nicola Ruperto; Anna Simon; Marinka Twilt; Joost Frenkel; Hal M Hoffman; Adriana A de Jesus; Jasmin B Kuemmerle-Deschner; Seza Ozen; Marco Gattorno; Raphaela Goldbach-Mansky; Erkan Demirkaya Journal: Arthritis Rheumatol Date: 2022-05-27 Impact factor: 15.483
Authors: Luciana B Paim-Marques; Amanda Cavalcante; Catherine Castro; Theresa L Wampler Muskardin; João Bosco de Oliveira; Timothy B Niewold; Simone Appenzeller Journal: Rheumatol Int Date: 2020-08-19 Impact factor: 2.631