Session Information
Session Type: Abstract Submissions (ACR)
Background/Purpose
The three phenotypes in the order of severity: 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), constitute the cryopyrin-associated periodic syndrome (CAPS).1 Here we present the interim safety data of canakinumab (CAN) use in CAPS patients (pts) in clinical practice from the on-going observational β–Confident Registry, that involves 40 sites in 13 countries. 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 pt enrollment (19 November 2009) until the current data cut-off date (15 April 2014). Additional safety data will be updated, as available, at the time of the presentation.
Results
272 pts (FCAS, n=38; MWS, n=164; NOMID, n=32; others, n=37) were enrolled with a mean±SD duration of 164±61.3 weeks in the registry. Of these, 17 (6.3%) pts discontinued CAN: 5 each due to AE, poor efficacy and patient preference; 2 for unknown reasons. The incidence rate of overall AEs was 100.0 per person-years (pyr). Pts with FCAS, the least severe phenotype, had the lowest AE incidence rate (49.1/100 pyr) as compared with pts with MWS (107.3/100 pyr) and NOMID (130.4/100 pyr), the more severe phenotypes. The most common types of AEs were infections and infestations with an incidence rate of 35.6/100 pyr. A total of 79 SAEs were reported by 47 pts resulting in an incidence rate of 13.2 SAEs/100 pyr. The most common type of SAE was infection (3.5/100 pyr). One death in a 76 yr MWS old pt with metastatic rectal adenocarcinoma was reported. Of 14 (5.1%) pts that received pneumococcal vaccination (PPV), 10 reported a local post-PPV injection site reaction; of which 4 were considered as serious. Based on PGA assessment, nearly half the pts had no disease activity while most others had mild/moderate disease activity, at the current data cut-off. There was no evidence of loss of effect with time.
Conclusion
The safety profile observed with the use of CAN in CAPS registry is consistent with that observed in the clinical trial program. Response rates were as expected based on clinical trial experience with no evidence of loss of efficacy.
1Farasat S et al. Arch Dermatol. 2008;144: 392-402
Disclosure:
H. M. Hoffman,
Novartis Pharmaceutical Corporation,
5;
J. B. Kuemmerle-Deschner,
Novartis ,
2,
Novartis ,
5;
P. N. Hawkins,
None;
T. van der Poll,
None;
U. A. Walker,
Novartis,
5;
K. Abrams,
Novartis Pharmaceutical Corporation,
3,
Novartis Pharmaceutical Corporation,
1;
H. H. Tilson,
Novartis ,
5.
« Back to 2014 ACR/ARHP Annual Meeting
ACR Meeting Abstracts - https://acrabstracts.org/abstract/canakinumab-use-in-patients-with-cryopyrin-associated-periodic-syndrome-interim-safety-and-efficacy-results-from-beta-confident-registry/