title: Real-life effectiveness of canakinumab in cryopyrin-associated periodic syndrome.
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%).
  
  
  CONCLUSIONS
  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.affiliation: Division of Pediatric Rheumatology, Department of Pediatrics, University Children's Hospital Tuebingen, kuemmerle.deschner@uni-tuebingen.de.
authors.forename: Jasmin B
authors.initials: JB
authors.lastname: Kuemmerle-Deschner
authors.affiliation: Division of Pediatric Rheumatology, Department of Pediatrics, University Children's Hospital Tuebingen.
authors.forename: Ferdinand
authors.initials: F
authors.lastname: Hofer
authors.affiliation: Division of Pediatric Rheumatology, Department of Pediatrics, University Children's Hospital Tuebingen.
authors.forename: Theresa
authors.initials: T
authors.lastname: Endres
authors.affiliation: Division of Nephrology, University of Marburg.
authors.forename: Birgit
authors.initials: B
authors.lastname: Kortus-Goetze
authors.affiliation: Haematology, Oncology and Rheumatology, University Hospital Heidelberg.
authors.forename: Norbert
authors.initials: N
authors.lastname: Blank
authors.affiliation: Pediatric Rheumatology Clinics, University Hospital Eppendorf, Hamburg.
authors.forename: Elisabeth
authors.initials: E
authors.lastname: Weißbarth-Riedel
authors.affiliation: Department of Pediatrics and Adolescents Medicine, University Hospital Ulm.
authors.forename: Catharina
authors.initials: C
authors.lastname: Schuetz
authors.affiliation: Childrens Hospital, Section Rheumatology, Charite Campus Virchow, Berlin.
authors.forename: Tilmann
authors.initials: T
authors.lastname: Kallinich
authors.affiliation: Allergie-Centrum Charite, Department for Dermatology, Charite Campus Mitte, Berlin.
authors.forename: Karoline
authors.initials: K
authors.lastname: Krause
authors.affiliation: Department of Rheumatology, Clementine Childrens Hospital, Frankfurt.
authors.forename: Christoph
authors.initials: C
authors.lastname: Rietschel
authors.affiliation: Centre for Pediatric Rheumatology Sankt Augustin, Asklepios Klinik Sankt Augustin, Sankt Augustin, Germany.
authors.forename: Gerd
authors.initials: G
authors.lastname: Horneff
authors.affiliation: Division of Pediatric Rheumatology, Department of Pediatrics, University Children's Hospital Tuebingen, and Rheumatology, Alberta Children's Hospital, University of Calgary, Alberta, Canada.
authors.forename: Susanne M
authors.initials: SM
authors.lastname: Benseler
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D000368:Aged; D000911:Antibodies, Monoclonal / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D061067:Antibodies, Monoclonal, Humanized; D002648:Child; D002675:Child, Preschool; D056587:Cryopyrin-Associated Periodic Syndromes / Q000188:drug therapy*; D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D053583:Interleukin-1beta / Q000037:antagonists & inhibitors; D008297:Male; D008875:Middle Aged; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D053583:Interleukin-1beta; C541220:canakinumab
keywords: CAPS; FCAS; MWS; Muckle–Wells syndrome; NOMID/CINCA; canakinumab; cryopyrin-associated periodic syndrome; effectiveness; outcome; treat-to-target