THU0392 Beta-confident-registry: Efficacy and safety of canakinumab in cryopyrin associated periodic syndrome - 18 month follow-up
Authors: J. Kuemmerle-Deschner, P. Hawkins, H. Hoffman, T. van der Poll, U. Walker, H. Tilson
Journal: Annals of the Rheumatic Diseases (2013)

<h3>Background</h3> 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. <h3>Objectives</h3> 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). <h3>Methods</h3> 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. <h3>Results</h3> 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). <h3>Conclusions</h3> 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. <h3>Disclosure of Interest</h3> 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