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SAT0524 Evaluation of Long-Term Safety and Effectiveness of Canakinumab Therapy in Patients with Cryopyrin-Associated Periodic Syndrome: Results from Beta-Confident Registry
  1. J. Kuemmerle-Deschner1,
  2. H. Hoffman2,
  3. P. Hawkins3,
  4. T. van der Poll4,
  5. U. Walker5,
  6. A. Speziale6,
  7. H. Tilson7
  1. 1University Hospital Tuebingen, Tuebingen, Germany
  2. 2University of California, La Jolla, CA, United States
  3. 3University College London Medical School, London, United Kingdom
  4. 4University of Amsterdam, Academic Medical Center, Amsterdam, Netherlands
  5. 5University Hospital
  6. 6Novartis Pharma AG, Basel, Switzerland
  7. 7University of North Carolina, Chapel Hill, United States


Background Cryopyrin-Associated Periodic Syndrome (CAPS), a rare hereditary autoinflammatory disorder consists of three groups/phenotypes: familial cold auto-inflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and chronic infantile neurologic cutaneous and articular syndrome/neonatal onset multisystem inflammatory disease (CINCA/NOMID).1 Here we evaluate the final interim data of canakinumab (CAN), a selective, human anti-IL-1β monoclonal antibody, use in CAPS patients in clinical practice from the multicentre, observational β–Confident Registry.

Objectives The primary objective is to monitor and explore the overall safety of CAN with a focus on serious adverse events (SAEs) including serious infections, vertigo, malignancies and hypersensitivity reactions. The secondary objective is to measure efficacy using physician global assessments (PGA).

Methods β–Confident Registry protocol does not mandate any visits or procedures, but records all observed and reported adverse events (AEs) and serious AE (SAEs) or AEs potentially related to treatment with CAN. Cumulative safety data are reported as incidence rate (number of events) per 100 patient-years (IR/100 pyr) from the date of first patient enrollment (19 November 2009) until the current data cut-off date (31 December, 2014). Additional safety data will be updated, as available, at the time of the presentation.

Results 288 patients (FCAS, n=40; MWS, n=167; NOMID, n=33; others, n=37) were enrolled with a mean ± SD duration of 193±72 weeks in the registry. Of these, 21 (7.3%) patients discontinued CAN: 5 each due to AE, poor efficacy, and patient preference; and 6 due to unknown reasons. The IR/100 pyr of overall AEs was 100.0. Patients with FCAS, the least severe phenotype, had the lowest AE IR/100 pyr (60.9) as compared with patients with MWS (IR/100 pyr 107.2) and NOMID (IR/100 pyr 120.3), the more severe phenotypes. The most common types of AEs were infections and infestations with an IR/100 pyr of 36.7. Vertigo was reported by 19 patients (IR/100 pyr 3.7). A total of 117 SAEs was reported by 62 patients resulting in an IR/100 pyr of 15.0 SAEs. The most common type of SAE was infection (IR/100 pyr 4.1). One death in a 76 yr old MWS patient with metastatic rectal adenocarcinoma was reported. Of 18 patients that received pneumococcal vaccination (PPV), 13 reported a local post-PPV injection site reaction; of which 4 were considered as serious. Based on PGA, nearly half the patients had no disease activity while most others had mild/moderate disease activity, at the current data cut-off. Similarly, disease activity was mostly absent in NLRP3 mutation negative CAPS patients (n=14) treated with CAN. There was no evidence of loss of effect with time.

Conclusions Long-term safety and effectiveness observed with the use of canakinumab in CAPS registry is consistent with that observed in the clinical trial program. Canakinumab therapy is also effective in NLRP3 mutation negative CAPS patients.


  1. Kuemmerle-Deschner JB, et al. Arthritis Res Ther. 2011;13(1):R34.

Disclosure of Interest J. Kuemmerle-Deschner Grant/research support from: Novartis, Consultant for: Novartis, H. Hoffman Consultant for: Novartis, Speakers bureau: Novartis, P. Hawkins: None declared, T. van der Poll: None declared, U. Walker Consultant for: Novartis, A. Speziale Employee of: Novartis, H. Tilson Consultant for: Novartis

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