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THU0536 Efficacy and Post-Vaccination Antibody Titer Data in Children with Caps Aged 28 Days to 4 Years Treated with Canakinumab
  1. Y. Uziel1,
  2. P. Brogan2,
  3. M. Hofer3,
  4. J. Kuemmerle-Deschner4,
  5. B. Lauwerys5,
  6. A. Speziale6,
  7. K. Abrams7,
  8. K. Leon7,
  9. X. Wei8,
  10. R. Laxer9,
  11. H. Lachmann10
  1. 1Pediatric Rheumatology Unit, Department of Pediatrics, Meir Medical Center, Kfar Saba, Israel
  2. 2Department of Paediatric Rheumatology, UCL Institute of Child Health, and Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom
  3. 3Unité romande de rhumatologie pédiatrique, Hospitalier Universitaire Vaudois, Lausanne, Switzerland
  4. 4University Hospital Tuebingen, Tuebingen, Germany
  5. 5Cliniques Universitaires Saint-Luc and Université catholique de Louvain, Brussels, Belgium
  6. 6Novartis Pharma AG, Basel, Switzerland
  7. 7Novartis Pharmaceuticals Corporation, New Jersey, United States
  8. 8Novartis Pharma, Beijing, China
  9. 9University of Toronto, Staff Rheumatologist, The Hospital for Sick Children, Toronto, Ontario, Canada
  10. 10National Amyloidosis Centre, UCL Medical School, London, United Kingdom


Background Canakinumab (CAN) has proven efficacy in patients with CAPS aged ≥2 years1. However, patients can require treatment in infancy where CAN has not yet been studied. IL-1 inhibition has not affected antibody production after vaccination in healthy volunteers2, but no data in CAPS patients receiving standard childhood vaccines are available.

Objectives To evaluate the efficacy and safety of CAN, including post-vaccination antibody production, in children with CAPS ≤4 yrs of age.

Methods CAN-naïve patients with confirmed CAPS aged 28 days to 4 yrs received open-label CAN dosed 2-12 mg/kg every 4 or 8 weeks for 56 weeks. Efficacy was evaluated by complete response (clinical response and normal CRP) and subsequent relapse. Safety was evaluated by adverse event (AE) reporting and vaccination response evaluated by post-vaccine antibody titers measured at 28 and 57 days post vaccination. Vaccines evaluated included DTP; H. Flu; N. Men.; influenza; Hep B; and Strep. Pneum.

Results Seventeen patients, 6 less than 24 months old (44 days-5 months), were enrolled. The phenotypic distribution was: FCAS (n=1), MWS (n=12), and NOMID (n=4). All 17 patients achieved a clinical response and 16 achieved a complete response. Seven patients required dose escalation to achieve and/or maintain their responses. The patient who did not achieve a complete response was a 1 yr old with persistently elevated CRP. Of the 16 with a complete response, 4 (2 with MWS and 2 with NOMID) subsequently relapsed, but all regained complete response; 2 (1 MWS; 1 NOMID) with and 2 (1 MWS; 1 NOMID) without dose escalation. No CAPS flares were reported with vaccination and a rise in post-vaccination antibody titers was observed for all vaccines evaluated. The most common type of AE reported was an infection, typically involving the upper respiratory tract. Four patients experienced a serious AE (SAE), with no SAE occurring more than once. No patient discontinued due to an AE.

Conclusions Canakinumab is a highly effective treatment for patients with CAPS aged as young as 44 days old. The safety profile was acceptable and similar to that observed for older patients. Canakinumab appears to have no effect on the ability to produce antibodies against standard childhood non-live vaccines.


  1. Lachmann H, et al. N Engl J Med. 2009;60:2416-25.

  2. Chioato A, et al. Clin Vaccine Immunol. 2010;17:1952-1957.

Disclosure of Interest Y. Uziel Consultant for: Novartis, Speakers bureau: Novartis, P. Brogan Grant/research support from: Institutional grant support to undertake the study described in this abstract, Consultant for: SOBI, Roche, M. Hofer Consultant for: Novartis, J. Kuemmerle-Deschner Grant/research support from: Novartis, Consultant for: Novartis, B. Lauwerys: None declared, A. Speziale Employee of: Novartis, K. Abrams Shareholder of: Novartis, Employee of: Novartis, K. Leon Employee of: Novartis, X. Wei Employee of: Novartis, R. Laxer Grant/research support from: Database funding from Novartis, Consultant for: Participant in Ad Board for Novartis, H. Lachmann Consultant for: Novartis, Speakers bureau: Novartis

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