Session Type: ACR Poster Session C
Session Time: 9:00AM-11:00AM
Background/Purpose: Cryopyrin-Associated Periodic Syndrome (CAPS), is a rare hereditary auto inflammatory disorder representing 3 phenotypes: 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).1 Canakinumab (CAN), a fully human anti–IL–1β monoclonal antibody, has been shown to be effective over 56 weeks in patients with CAPS aged <24 months (core study).2 Here we report the long-term efficacy and safety of canakinumab in these patients through 152 weeks in an extension study to the core study (NCT01576367).
Methods: CAPS pts who completed the core study received 2 mg/kg subcutaneous CAN every 8 weeks, in continuation with the core study. Pts who received a dose adjustment in the core study were continued on the same dose in the extension phase. Efficacy was evaluated by investigator clinical assessment of autoinflammatory disease activity, C-reactive protein (CRP) and serum amyloid A (SAA) levels. Safety was assessed in terms of adverse events (AEs).
Results: Of 17 pts (aged ≤6 years), enrolled in the extension study, 12 (70.6%), 4 (23.5%) and 1 (5.9%) had MWS, NOMID and FCAS phenotypes, respectively. All 17 pts were complete responders; 16 (94.1%) had no flare, and 1 (5.9%) NOMID patient had a flare at the end of extension study. Physician global assessment improved over the extension study, with decline in severity from baseline of the core study to the end of extension study (EOS). The number of pts with absent autoinflammatory disease activity improved from 4 (23.5%) to 11 (64.7%); minimal activity increased from 5.9% (1 pt) to 29.4% (5 pts); mild or moderate activity decreased from 47.1% (8 pts) to 5.9% (1 pt); moderate activity decreased from 23.5% (4 pts) to 0 patients. This improvement was also observed in the assessment of skin rash; proportion of patients with no skin disease increased from 29.4% (5 pts) at baseline of core study to 94.1% (16 pts) at EOS. The mean decrease in CRP and SAA levels from core study baseline was −10.4 and −54.36 mg/L, respectively at EOS. Overall, 10 (58.8%) pts had AEs suspected to be related to CAN; the most common were diarrhea, pneumonia, rhinitis and cough (3 pts each). Eight (47.1%) pts experienced at least 1 serious AE (4 MWS and 4 NOMID pts), with pneumonia being the most common (2 [11.8%]). No deaths occurred during the study.
Conclusion: Canakinumab effectively maintained clinical and serological efficacy in CAPS pts in the extension study. No new safety findings were observed, and the safety profile of canakinumab was consistent with previous studies, which corroborates the long-term use of canakinumab in the treatment of CAPS patients. References: 1. Kuemmerle-Deschner JB, et al. Arthritis Res Ther. 2011;13(1):R34. 2. Brogan, P et al. Arthritis Rheumatol.2015;67(suppl 10).
To cite this abstract in AMA style:Brogan P, Hofer M, Kuemmerle-Deschner JB, Lauwerys B, Speziale A, Leon K, Wei X, Laxer R. Efficacy and Safety of Canakinumab in Patients Aged One to Six Years with Cryopyrin-Associated Periodic Syndromes: Results of an Open-Label, Phase III Extension Study [abstract]. Arthritis Rheumatol. 2016; 68 (suppl 10). https://acrabstracts.org/abstract/efficacy-and-safety-of-canakinumab-in-patients-aged-one-to-six-years-with-cryopyrin-associated-periodic-syndromes-results-of-an-open-label-phase-iii-extension-study/. Accessed May 21, 2019.
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ACR Meeting Abstracts - https://acrabstracts.org/abstract/efficacy-and-safety-of-canakinumab-in-patients-aged-one-to-six-years-with-cryopyrin-associated-periodic-syndromes-results-of-an-open-label-phase-iii-extension-study/