Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry
Objective To report the long-term safety and effectiveness of canakinumab, a fully human anti-interleukin 1β monoclonal antibody, in patients with cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS) and neonatal-onset...
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| Format: | Article |
| Language: | English |
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BMJ Publishing Group
2021-08-01
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| Series: | RMD Open |
| Online Access: | https://rmdopen.bmj.com/content/7/2/e001663.full |
| _version_ | 1831570558573084672 |
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| author | Ulrich A Walker Jasmin B Kuemmerle-Deschner Jeremy Levy Eleni Vritzali Hal M Hoffman Tom van der Poll PHILIP N HAWKINS Hugh H Tilson Stephanie Noviello |
| author_facet | Ulrich A Walker Jasmin B Kuemmerle-Deschner Jeremy Levy Eleni Vritzali Hal M Hoffman Tom van der Poll PHILIP N HAWKINS Hugh H Tilson Stephanie Noviello |
| author_sort | Ulrich A Walker |
| collection | DOAJ |
| description | Objective To report the long-term safety and effectiveness of canakinumab, a fully human anti-interleukin 1β monoclonal antibody, in patients with cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS) and neonatal-onset multisystem inflammatory disease (NOMID), in a real-world setting.Methods From December 2009 to December 2015, the β-Confident Registry prospectively enrolled patients with CAPS and non-CAPS conditions who received canakinumab per routine care and were prospectively followed for up to 6 years. The registry protocol did not mandate specific visits or procedures; however, all observed adverse events (AEs) and serious adverse events (SAEs) had to be recorded. Canakinumab effectiveness was evaluated by Physician’s Global Assessment (PGA).Results Of 288 patients enrolled, 3 were excluded due to missing informed consent. Among the remaining 285 patients, 243 (85.3%) were patients with CAPS and 42 (14.7%) had atypical CAPS (6.3%) or other conditions (8.4%). The median age was 26.6 years. Based on PGA, 58 of 123 (47.2%) patients with CAPS had no disease activity at 48 months, and 65 of 123 (52.8%) experienced mild/moderate disease activity at 48 months. Among CAPS phenotypes, AE incidence rates per 100 patient-years were lowest for FCAS (73.1; 95% CI 60.3 to 87.8) compared with those with MWS (105.0; 95% CI 97.2 to 113.2) or NOMID (104.6; 95% CI 86.6 to 125.2). One hundred twenty-eight SAEs were reported in 68 patients with CAPS (incidence rate/100 patient-years, 14.0; 95% CI 11.6 to 16.6). One death (metastatic rectal adenocarcinoma in a patient with MWS) was reported.Conclusions The response to canakinumab was sustained for up to 6 years. Canakinumab demonstrated a favourable safety profile over long-term treatment in patients with CAPS.Trial registration number NCT01213641. |
| first_indexed | 2024-12-17T12:53:22Z |
| format | Article |
| id | doaj.art-5a7617904f374234acba1f6d7e2849d6 |
| institution | Directory Open Access Journal |
| issn | 2056-5933 |
| language | English |
| last_indexed | 2024-12-17T12:53:22Z |
| publishDate | 2021-08-01 |
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| series | RMD Open |
| spelling | doaj.art-5a7617904f374234acba1f6d7e2849d62022-12-21T21:47:33ZengBMJ Publishing GroupRMD Open2056-59332021-08-017210.1136/rmdopen-2021-001663Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident RegistryUlrich A Walker0Jasmin B Kuemmerle-Deschner1Jeremy Levy2Eleni Vritzali3Hal M Hoffman4Tom van der Poll5PHILIP N HAWKINS6Hugh H Tilson7Stephanie Noviello8Department of Rheumatology, University Hospital Basel, Basel, SwitzerlandDivision of Pediatric Rheumatology, Department of Paediatrics and Autoinflammation Reference Center Tuebingen, University Hospital Tuebingen, Tübingen, GermanyNovartis Pharma AG, Basel, SwitzerlandNovartis Pharma AG, Basel, SwitzerlandUniversity of California San Diego, San Diego, California, USAAmsterdam Medical Center, University of Amsterdam, Amsterdam, The NetherlandsUniversity College London, London, UKGillings School of Global Public Health, University of North Carolina, Chapel Hill, North Carolina, USANovartis Pharmaceuticals Corporation, East Hanover, New Jersey, USAObjective To report the long-term safety and effectiveness of canakinumab, a fully human anti-interleukin 1β monoclonal antibody, in patients with cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS) and neonatal-onset multisystem inflammatory disease (NOMID), in a real-world setting.Methods From December 2009 to December 2015, the β-Confident Registry prospectively enrolled patients with CAPS and non-CAPS conditions who received canakinumab per routine care and were prospectively followed for up to 6 years. The registry protocol did not mandate specific visits or procedures; however, all observed adverse events (AEs) and serious adverse events (SAEs) had to be recorded. Canakinumab effectiveness was evaluated by Physician’s Global Assessment (PGA).Results Of 288 patients enrolled, 3 were excluded due to missing informed consent. Among the remaining 285 patients, 243 (85.3%) were patients with CAPS and 42 (14.7%) had atypical CAPS (6.3%) or other conditions (8.4%). The median age was 26.6 years. Based on PGA, 58 of 123 (47.2%) patients with CAPS had no disease activity at 48 months, and 65 of 123 (52.8%) experienced mild/moderate disease activity at 48 months. Among CAPS phenotypes, AE incidence rates per 100 patient-years were lowest for FCAS (73.1; 95% CI 60.3 to 87.8) compared with those with MWS (105.0; 95% CI 97.2 to 113.2) or NOMID (104.6; 95% CI 86.6 to 125.2). One hundred twenty-eight SAEs were reported in 68 patients with CAPS (incidence rate/100 patient-years, 14.0; 95% CI 11.6 to 16.6). One death (metastatic rectal adenocarcinoma in a patient with MWS) was reported.Conclusions The response to canakinumab was sustained for up to 6 years. Canakinumab demonstrated a favourable safety profile over long-term treatment in patients with CAPS.Trial registration number NCT01213641.https://rmdopen.bmj.com/content/7/2/e001663.full |
| spellingShingle | Ulrich A Walker Jasmin B Kuemmerle-Deschner Jeremy Levy Eleni Vritzali Hal M Hoffman Tom van der Poll PHILIP N HAWKINS Hugh H Tilson Stephanie Noviello Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry RMD Open |
| title | Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry |
| title_full | Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry |
| title_fullStr | Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry |
| title_full_unstemmed | Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry |
| title_short | Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry |
| title_sort | long term safety and effectiveness of canakinumab therapy in patients with cryopyrin associated periodic syndrome results from the β confident registry |
| url | https://rmdopen.bmj.com/content/7/2/e001663.full |
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