Session Title: Miscellaneous Rheumatic and Inflammatory Diseases - Poster I
Session Type: ACR Poster Session A
Session Time: 9:00AM-11:00AM
Background/Purpose: Cryopyrin-associated periodic syndrome (CAPS) is a rare auto-inflammatory disease encompassing a spectrum of 3 phenotypes: familial cold auto-inflammatory syndrome (FCAS), Muckle–Wells syndrome (MWS), and neonatal-onset multisystem inflammatory disease (NOMID).1 The estimated frequency of CAPS ranges from 1 to 3 per million.2,3 The β-Confident Registry (NCT01213641) is a multicenter, long-term, prospective, observational study with an enrollment period of 5 years (yrs) to monitor the long-term safety and efficacy of canakinumab (CAN) for CAPS. Here we report the overall final results. The primary objective of the Registry was to monitor the safety of CAN, focusing on serious adverse events (SAEs) including serious infections, vertigo, malignancies, and hypersensitivity reactions.
Methods: : Cumulative safety data were reported as incidence rate per 100 patient-yrs (IR/100 pyrs) from the enrollment of the first patient (November 2009) until end of study (December 2015). After enrollment patients were followed up for at least 1 yr. The Registry protocol did not mandate any visits or procedures; however, all observed and reported AEs and SAEs or AEs potentially related to CAN were recorded. Efficacy was measured using physician’s global assessment (PGA).
Results: Overall, 288 patients (FCAS, n=42; MWS, n=170; NOMID, n=34; others, n=42) were enrolled at 39 sites across 13 countries, with a mean patient exposure duration of 3.6 yrs. Of these, 22 (8.0%) discontinued CAN: 5 due to AEs, 10 due to poor efficacy and patient preference, and 7 due to other reasons. The IR/100 pyrs for overall AEs was 100.0. Among the phenotypes of CAPS, patients with FCAS had the lowest IR/100 pyrs (78.1) than patients with MWS (113.4), and NOMID (119.0). The most common AEs were infections and infestations (IR/100 pyrs, 39.1). Overall, 161 SAEs were reported by 86 patients (IR/100 pyrs, 16.3), most commonly, infections (IR/100 pyrs, 5.0). One death (metastatic rectal adenocarcinoma in a 76-yr-old MWS patient) was reported. Of 23 patients who received pneumococcal vaccination (PPV), 15 (65.0%) reported local post-PPV injection site reactions, of which 5 were considered serious. Based on PGA, 31.0% of patients had no disease activity, whereas most (59.0%) of the others experienced mild/moderate disease activity at Month 54. Similarly, disease activity was absent or mild/moderate in patients with NLRP3 mutation-negative CAPS (n=13) treated with CAN.
Conclusion: The β-confident Registry is the largest CAPS cohort documented in a registry. This Registry showed that the safety profile of canakinumab was consistent with previous findings and efficacy was sustained for more than 5 years. Canakinumab therapy was also effective in patients with NLRP3 mutation-negative CAPS. References:
- Kuemmerle-Deschner JB, et al. Arthritis Res Ther. 2011;13(1):R34.
- Cuisset L, et al. Ann Rheum Dis. 2011;70(3):495-9.
- Tilson H, et al. Orphanet J Rare Dis. 2013;10;8:139.
To cite this abstract in AMA style:Hoffman HM, Kuemmerle-Deschner JB, Hawkins PN, van der Poll T, Walker UA, Speziale A, Joubert Y, Tilson HH. Safety and Efficacy of Long-Term Canakinumab Therapy in Patients with CAPS: Final Results from Beta-Confident Registry [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/safety-and-efficacy-of-long-term-canakinumab-therapy-in-patients-with-caps-final-results-from-beta-confident-registry/. Accessed December 2, 2020.
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