Session Information
Session Type: Abstract Submissions (ACR)
Background/Purpose: Cryopyrin-associated periodic syndrome (CAPS) comprises s extremely rare auto-inflammatory disorders, including familial cold auto-inflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and neonatal-onset multisystem inflammatory disease/chronic infantile neurological cutaneous and articular syndrome (NOMID/CINCA).1 NLRP3 gene mutation results in overproduction of IL-1β, leading to observed clinical symptoms of CAPS.2 Canakinumab (CAN) is a selective anti-IL-1β monoclonal antibody, approved for the treatment of CAPS.2,3 Here, we report the updated safety data for CAN in CAPS patients (pts) from the ongoing β-Confident Registry. The primary objective of the Registry is to monitor and further explore the overall safety of CAN, focusing primarily on serious infections, malignancies, hypersensitivity reactions, vertigo, and other selected adverse events in CAPS pts over a 5-year period (NCT01213641).
Methods: The Registry includes pts with CAPS and other auto-inflammatory disorders receiving CAN as part of regular medical care, after obtaining the written informed consent The study protocol does not mandate any visits or procedures, records all observed and reported adverse events (AEs) and serious adverse events (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 (November 19, 2009) until the data cut-off date (March 29, 2013). Additional safety data will be updated, as available, at the time of the conference presentation.
Results: A total of 245 pts were enrolled in the Registry at the current cut-off date, of which 229 patients reported a median duration of 50 weeks (range: 0.9 – 137.3) of prior treatment with CAN at the time of Registry enrollment. Of the total, 100 pts reported 238 AEs with an IR of 75.9/100 pyr. Infections with an IR of 20.7/100 pyr, were the most commonly observed AEs and included nasopharyringitis, rhinitis and urinary tract infections (UTI) among others. Eight pts reported 13 events of vertigo as AEs, resulting in an IR of 4.2/100 pyr, of which 8 were suspected to be CAN related. With regards to SAEs, 37 events were reported by 27 pts with an IR of 11.8 /100 pyr that included one fatal case of malignancy (metastatic rectal adenocarcinoma in a 76 yr MWS patient) and 12 events of serious infections. No case of hypersensitivity to CAN was reported. A total of 14 pts permanently discontinued CAN: 4 each due to pts preference and AEs, 3 due to lack of therapeutic effects, and 3 due to unspecified reasons.
Conclusion: No new or unexpected safety signals were reported to date in this ongoing Registry. Infections, as expected, were the most commonly occurring AEs and SAEs.
References: 1. Arthritis Res Ther 2011, 13:R34, 2. Lachmann et al. J Exp Med 2009, 206:1029-1036, 3. Arthritis Rheum 2008;58:2443–2452
Disclosure:
H. Hoffman,
Novartis, Regeneron, Sobi Biovitrum,
5;
J. B. Kuemmerle-Deschner,
Novartis,,
2,
Novartis,,
5;
P. N. Hawkins,
None;
T. van der Poll,
Novartis,,
5;
U. A. Walker,
Novartis,,
5;
M. Nebesky,
Novartis,,
3;
K. Abrams,
Novartis,,
3,
Novartis, ,
1;
H. Tilson,
Bio Soteria, Bristol-Myers Squibb, Gilead,GlaxoSmithKline, HealthCore, Kendle, Merck, Novartis,
5,
Glaxo SmithKline, Procter & Gamble,Other non-pharmaceutical holdings,
1.
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