Arthritis & Rheumatism, Volume 62,
November 2010 Abstract Supplement

Abstracts of the American College of
Rheumatology/Association of Rheumatology Health Professionals
Annual Scientific Meeting
Atlanta, Georgia November 6-11, 2010.

Canakinumab (ILARIS) Provides Rapid Response and Sustained Remission in Children across Different Disease Severity Phenotypes of Cryopyrin Associated Periodic Syndrome (CAPS).

Quartier13,  P., Hachulla2,  E., Gattorno5,  M., Cartwright1,  R., Kone-Paut4,  I., Zulian15,  F., Weisbarth-Riedel6,  E.

Allergy Center at Brookstone, Columbus, GA
S.C.U. Clinica Pediatrica, IRCCS Burlo Garofolo, Trieste, Italy
UCSF School of Medicine, San Francisco, CA
Universitaetsklinikum Giessen und Marburg GmbH, Marburg, Germany
Universite Paris-Descartes and Unite d'Immuno-Hematologie et Rhumatologie Pediatrique, Necker-Enfants Malades, Assistance Publique Hopitaux de Paris, Paris, France
University College London Medical School, London, UK
University of Padova School of Medicine, Padova, Italy
Zentrum für Reumatologie, Hamburger Zentrum fuer Kinder-und Jugendrheumatologie, Hamburg, Germany
Department of Internal Medicine, Hôpital Claude Huriez CHRU, Lille Cedex, France
Department of Pediatrics, Hospital Central de Asturias, Oviedo, Spain
Hôpital Kremlin Bicetre, CEREMAI, Le Kremlin Bicetre, France
Istituto Giannina Gaslini, Genova, Italy
Kinderklinik, Rheum. Ambulanz, Universitätsklinikum Hamburg-Eppendorf, Hamburg, Germany
Klinik fuer Kinder-und Jugendmedizin, Universitaetsklinikum, Tuebingen, Germany
Novartis Pharma AG, Basel, Switzerland
Novartis Pharmaceuticals Corporation, East Hanover, NJ


Canakinumab provides sustained interleukin-1b (IL-1b) blockade and is effective in the treatment of CAPS (comprising of familial cold auto-inflammatory syndrome [FCAS], Muckle-Wells syndrome [MWS], chronic infantile neurologic, cutaneous and articular syndrome [CINCA]/neonatal-onset multisystem inflammatory disease [NOMID]). Herein we report the long-term safety, tolerability, and efficacy of canakinumab in the subgroup of pediatric CAPS patients who were enrolled as part of an open-label, single-treatment arm, Phase III study.


Patients enrolled in this study were canakinumab-naive or rolled-over from earlier Phase II/III studies. Patients received canakinumab 150 mg s.c. or 2 mg/kg s.c. (body weight <=40 kg) every 8 weeks. Complete response was assessed for canakinumab-naive patients, while roll-over patients entered the study allowing continuous treatment every 8 weeks. In case of an incomplete response patients could be changed to a more intense dosing regimen (received an additional dose of canakinumab 300 mg s.c. or 4 mg/kg s.c. [body weight <=40 kg]). Adjustment of the dosing frequency was also permissible. Complete response was defined as: physician's global assessment of disease activity and skin assessment score <=minimal and normal CRP and/or SAA values (<10 mg/L). Relapse was defined as: serum levels of CRP and/or SAA >30 mg/L and (a) physician's global assessment of disease activity >minimal or (b) physician's global assessment of disease activity minimal along with the assessment of skin disease >minimal.


Of 47 pediatric patients (3–17 years) (1 patient did not have CAPS and was discontinued [protocol violation] and of the remaining 46 patients there were 5 FCAS, 23 MWS, 18 MWS/NOMID [includes 8 NOMID] patients), 38 were canakinumab-naive, while 9 had been pre-treated with canakinumab. 43 (92%) completed the study and 3 patients discontinued the study (1 due to adverse event [AE]). The median duration of exposure to study drug was 290 days (range: 29–625 days). Complete response was achieved in most (n=26, 68%) of canakinumab-naive pediatric patients. The majority of canakinumab treated pediatric patients were relapse free (29 out of 35 [83%]) and 6 patients experienced a relapse. Dose adjustments were required in 16 patients (34%). Amongst all NOMID patients (including MWS/NOMID overlap) 15 (47%) received at least one dose or frequency adjustment. In canakinumab-naive patients median CRP and SAA levels rapidly decreased within 7 days (2.5 and 7.4 mg/L [baseline levels were 14.8 and 40.6 mg/L, respectively]) and these levels were maintained at normal levels (<10 mg/L) during the study (both in naive and roll-over patients). The most frequent AEs were rhinitis, nasopharyngitis and headache. Serious AEs were reported in 6 pediatric patients. Most patients (n=43, 91%) had no injection site reactions.


Canakinumab s.c. every 8 weeks induced rapid and sustained clinical and biochemical remission in pediatric patients across all severity of CAPS phenotypes. Adjustment of dose appears to be predominantly applied in pediatric and/or NOMID patients. Long-term tolerability and safety was good.

To cite this abstract, please use the following information:
Quartier, P., Hachulla, E., Gattorno, M., Cartwright, R., Kone-Paut, I., Zulian, F., et al; Canakinumab (ILARIS) Provides Rapid Response and Sustained Remission in Children across Different Disease Severity Phenotypes of Cryopyrin Associated Periodic Syndrome (CAPS). [abstract]. Arthritis Rheum 2010;62 Suppl 10 :1435
DOI: 10.1002/art.29201

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