Article
Beta-Confident Registry Results Show Long-Term Safety And Efficacy Of Canakinumab in Cryopyrin-Associated Periodic Syndrome (CAPS) Patients
Search Medline for
Authors
-
Jasmin Kümmerle-Deschner
- Universitätskinderklinik Tuebingen, Rheumatologie, Tübingen
-
Harold M. Hoffman
- University of California at San Diego, San Diego, United States,, San Diego, United States of America
-
Philip N. Hawkins
- University College London, Medical School, London, England
-
Tom van der Poll
- Academic Medical Center, University of Amsterdam, Amsterdam, Netherlands, Amstedam
-
Ulrich A. Walker
- Universitäts-Poliklinik, Felix-Platter Spital, Basel, Schweiz
-
Antonio Speziale
- Novartis Pharma AG, Immunologie & Dermatologie, Basel, Switzerland
-
Hugh Tilson
- University of North Carolina, School of Public Health, Chapel Hill, United States of America
| Published: | September 1, 2015 |
|---|
Outline
Text
Introduction: The β-Confident Registry, the largest CAPS cohort documented in a registry, enrolled the last patient in December 2014. Here, we report interim data for the complete cohort of enrolled patients. The objective is to monitor the overall safety of canakinumab (CAN) focusing on SAEs including serious infections, vertigo, malignancies, and hypersensitivity reactions.
Methods: The registry protocol does not mandate any visits or procedures, but records all observed and reported AEs and SAEs or AEs potentially CAN-related. Cumulative safety data are reported as incidence rate per 100 patient-years (IR/100 pyr). Data is partial for 11 patients due to the cut-off date for the analysis and will be updated at a later date. Efficacy was measured using physician global assessments (PGA).
Results: 288 patients were enrolled with a mean±SD duration of 193±72 weeks. 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 for overall AEs was 100.0. FCAS patients had the lowest AE IR/100 pyr (60.9) compared with MWS (IR/100 pyr 107.2) and NOMID (IR/100 pyr 120.3) patients. The most common types of AEs were infections and infestations (IR/100 pyr 36.7). Vertigo was reported by 19 patients (IR/100 pyr 3.7). 117 SAEs were reported by 62 patients (IR/100 pyr 15.0), with infection being the most common (IR/100 pyr 4.1). One death (metastatic rectal adenocarcinoma in 76 yr old MWS patient) was reported. Of 18 patients receiving pneumococcal vaccinations (PPV), 13 (72%) reported a local post-PPV injection site reaction, of which 5 were considered as serious. Based on PGA, nearly half the patients had no disease activity while most others had mild/moderate disease activity. 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. Further analyses of this cohort are ongoing.
Conclusion: Canakinumab demonstrated a safety profile consistent with that observed in the clinical trial program and provided continued effectiveness in CAPS patients for up to 5 years. Canakinumab therapy was also effective in NLRP3 mutation negative CAPS patients.
