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THU0392 Beta-confident-registry: Efficacy and safety of canakinumab in cryopyrin associated periodic syndrome - 18 month follow-up
  1. J. Kuemmerle-Deschner1,
  2. P. Hawkins2,
  3. H. Hoffman3,
  4. T. van der Poll4,
  5. U. Walker5,
  6. H. Tilson6
  1. 1Division of Pediatric Rheumatology, Department of Pediatrics, University Hospital Tuebingen, Tuebingen, Germany
  2. 2University College London Medical School, London, United Kingdom
  3. 3Division of Rheumatology and Allergy/Immunology, University of California at San Diego, San Diego, United States
  4. 4Academic Medical Center, University of Amsterdam, Amsterdam, Netherlands
  5. 5Universitäts-Poliklinik, Felix-Platter Spital, Basel, Switzerland
  6. 6University of North Carolina, School of Public Health, Chapel Hill, NC, United States


Background Canakinumab (CAN, Ilaris®), a fully human anti-IL-1β monoclonal antibody, selectively inhibits IL-1β and has been approved for the treatment of cryopyrin-associated periodic syndrome (CAPS), an extremely rare inherited inflammatory disorder.

Objectives The β-Confident Registry study (Registry) is a global observational study to monitor CAN safety profile and assess growth, development pattern and disease progression over 5 years in a larger cohort of CAPS patients (pts).

Methods CAPS pts receiving CAN as part of their routine care are included in this study. Primary objective of the Registry is to monitor the long-term safety and effectiveness of CAN. The Registry is updated with the data collected from routine clinical assessments in every 6 months (M). This abstract presents results of physician’s global assessment of auto-inflammatory disease activity, C-reactive protein (CRP) and serum amyloid A (SAA) levels and safety (adverse events [AEs] and serious AEs [SAEs]) of pts at 18M.

Results The registry was launched in Dec 2009 and has enrolled 177 pts (88 male/89 female; 129 age ≥18 years) with CAPS (23 FCAS, 105 MWS, 12 NOMID, 18 other inflammatory diseases) over a period of 18M. Median treatment duration was 87 weeks (Wk) (4.3-121.7; FACS: 87.0Wk [56.4-108.6]; MWS: 78.3Wk [4.3-121.7]; NOMID: 84.9Wk [26.1-104.3]; others 91.4Wk [13.0-121.7]) and mean number of injections/pt was 6.0±4.5 (FACS: 3.8±3.2; MWS: 6.2±4.1; NOMID: 7.2±3.7; others: 7.5±6.7). Auto-inflammatory disease activity (physician’s global assessment) was present in 2 pts (1.1%) at M18, compared to 63 pts (35.6%) at baseline (BL). Absence of auto-inflammatory disease activity was higher in CAN pretreated group than CAN naïve group (44.9% vs 21.7%) at BL. At M18, CRP and SAA levels decreased significantly (3.0±1.5 and 10.0±7.1 mg/L) vs BL (11.1±24.6 and 23.1±44.5 mg/L, respectively). The decrease in CRP level from BL to M18 was more prominent in pts pretreated with CAN vs CAN naïve pts (10.9±25.6 vs 3.0±1.5 mg/L and 11.9±20.5 vs 6.0±1.7 mg/L, respectively). At M12, SAA levels for both groups were comparable (15.2±26.9 vs 12.7±17.6 mg/L). A total of 32 AEs were reported by 17 pts (9.6%), most frequent AEs were infections reported in 7 pts (4%), (1 [0.6%] each of erysipelas, otitis media, rhinitis, urinary tract infection; 2 each [1.1%] of nasopharyngitis and pharyngitis), followed by ear and labyrinth disorders (vertigo) reported in 3 pts (1.7%). SAEs were reported in 4 (2.3%) pts (1 each of erysipelas, decrease in hemoglobin, ventricular tachycardia and 1 was not coded).

Conclusions Safety and efficacy profile of CAN during this 18M follow-up is similar to earlier data and no unexpected safety events were reported. CAN effectively suppressed inflammation in these CAPS pts and was well tolerated.

Disclosure of Interest J. Kuemmerle-Deschner Grant/Research support from: Novartis, P. Hawkins Grant/Research support from: Novartis, H. Hoffman Grant/Research support from: Novartis, T. van der Poll Grant/Research support from: Novartis, U. Walker Grant/Research support from: Novartis, H. Tilson Grant/Research support from: Novartis

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