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FRI0503 Efficacy and Safety of Canakinumab in Patients with Cryopyrin Associated Periodic Syndromes: An Open-Label, Phase-III, Extension Study
  1. P.A. Brogan1,
  2. M. Hofer2,
  3. J.B. Kuemmerle-Deschner3,
  4. B. Lauwerys4,
  5. A. Speziale5,
  6. K. Leon6,
  7. X. Wei7,
  8. R. Laxer8
  1. 1Department of Infection Inflammation and Rheumatology, UCL Institute of Child Health, and Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom
  2. 2Unité Romande de Rhumatologie Pédiatrique, Hospitalier Universitaire Vaudois, Lausanne, Switzerland
  3. 3University Hospital Tuebingen, Tuebingen, Germany
  4. 4Cliniques Universitaires Saint Luc and Université Catholique de Louvain, Brussels, Belgium
  5. 5Novartis Pharma AG, Basel, Switzerland
  6. 6Novartis Pharmaceuticals Corporation, New Jersey, United States
  7. 7Novartis Pharma, Beijing, China
  8. 8University of Toronto, The Hospital for Sick Children, Toronto, Ontario, Canada


Background Cryopyrin-Associated Periodic Syndrome (CAPS), a rare hereditary auto inflammatory disorder, constitutes 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 monoclonal anti-human interleukin-1β (IL-1β) antibody is indicated for the treatment of CAPS patients (either ≥2 years or ≥4 years with body weight ≥7.5 kg.2 Here, we present longer-term efficacy and safety of CAN in patients with CAPS aged >24 months.

Objectives To assess long-term efficacy of CAN with respect to relapse in CAPS patients who completed the core (CACZ885D2307) study

Methods CAPS pts (aged ≥1 years), who completed the core study, received a starting dose of 2 mg/kg s.c. of CAN every 8 weeks, in continuation with the core study. Patients who received a dose adjustment in the core study were allowed to continue same dose in the extension trial. Efficacy was evaluated by physician's global assessment (PGA), C-reactive protein (CRP) and serum amyloid A (SAA) levels. Safety was assessed in terms of adverse events (AEs).

Results Of 17 pts enrolled, 12 (70.6%), 4 (23.5%) and 1 (5.9) were of MWS, NOMID and FCAS phenotypes, respectively. All 17 pts were complete responders; 16 (94.1%) had no relapse and 1 (5.9%) NOMID patient had a relapse at the end of study (EOS). PGA improved over the extension study with decline in severity till EOS. The number of pts with absent autoinflammatory disease improved from 23.5% (4 pts) to 64.7% (11 pts). This improvement was also observed in the assessment of skin rash; proportion of patients with absent skin disease increased from 29.4% (5 pts) to 94.1% (16 pts). The mean decrease in CRP and SAA levels from baseline were -10.4mg/L and -54.36/L respectively. Overall, 10 (58.8%) pts had AEs suspected to be related to study drug; the most common were diarrhoea, 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 were reported during the study.

Conclusions CAN effectively maintained clinical and serological efficacy in CAPS pts. No new safety findings were observed and the safety profile of CAN was consistent with previous studies, which corroborates the long-term use of CAN in the treatment of CAPS patients.

  1. Kuemmerle-Deschner JB, et al. Arthritis Res Ther. 2011;13(1):R34.

  2. ILARIS (Package Insert). Novartis Pharmaceuticals, Inc. 2014

Disclosure of Interest P. Brogan Grant/research support from: Novartis Roche and SOBI, Consultant for: Novartis Roche and SOBI, M. Hofer Consultant for: Novartis, J. Kuemmerle-Deschner Grant/research support from: Novartis, Consultant for: Novartis, SOBI, Baxalta, B. Lauwerys: None declared, A. Speziale Employee of: Novartis, K. Leon Employee of: Novartis, X. Wei Employee of: Novartis, R. Laxer Grant/research support from: Database funding from Novartis

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