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) Improves Health-Related Quality of Life (HRQoL) in Patients with Cryopyrin-Associated Periodic Fever Syndrome (CAPS): Results of a Phase III, Open-Label Study in a Large Cohort of CAPS Patients.

Kone-Paut4,  I., Lachmann12,  H. J., Kuemmerle-Deschner6,  J. B., Hachulla3,  E., Cartwright1,  R., Hoyer2,  J., Quartier11,  P.

Allergy Center at Brookstone, Columbus, GA
UCSF School of Medicine, San Francisco, CA
Unit'e d'Immunologie, Hematologie et Rhumatologie Pediatrique, Hopital Necker-Enfants Malades, Paris, France
University College London Medical School, London, United Kingdom
University of Wisconsin Hospital and Clinics, Madison, WI
Department of Internal Medicine and Nephrology, Universitaetsklinikum Giessen und Marburg GmbH, Marburg, Germany
Hôpital Claude Huriez CHRU, Lille Cedex, France
Hôpital Kremlin Bicetre, CEREMAI, Le Kremlin Bicetre, France
Istituto Giannina Gaslini, Genova, Italy
Klinik fuer Kinder-und Jugendmedizin, Universitaetsklinikum, Tuebingen, Germany
Novartis Pharma AG, Basel, Switzerland
Novartis Pharmaceuticals Corporation, East Hanover, NJ
Rheumazentrum Ruhrgebiet, Herne, Germany

Background:

Canakinumab (a fully human anti-interleukin-1b monoclonal antibody) has been recently approved by the FDA for the treatment of familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS) and by the EU for the treatment of cryopyrin-associated periodic syndrome (CAPS). This spectrum of autoinflammatory disease is caused by overproduction of IL-1b and is associated with rash, constitutional inflammatory symptoms and marked fatigue. Health-related quality of life (HRQoL) was assessed during a phase III, open-label, multi-center study to evaluate the direct impact of the disease on patients' well-being and to assess the long-term maintenance of HRQoL.

Methods:

The 119 adult CAPS patients in this study were canakinumab-naive (n=71) or rolled-over from earlier Phase II/III studies (n=48). They received canakinumab 150 mg s.c. every 8 weeks and HRQoL was assessed using the following domains: general physical and mental health (SF-36® physical and mental component summary [PCS and MCS]), fatigue (FACIT-Fatigue©) and functional disability (HAQ-DI).

Results:

Median duration of exposure to canakinumab in the study was 414 days (range 29–687 days). Of the 119 adult CAPS patients there were 25 FCAS, 80 MWS, 14 MWS/NOMID [6 NOMID] patients. In canakinumab-naive patients the baseline scores (mean [SD]) for physical function (SF-36® PCS: 41.2 [10.9]) and mental health (SF-36® MCS: 45.5 [10.7]) and fatigue (FACIT-F: 32.8 [11.7]) were considerably lower than those expected in the general population (50 [10] for SF-36® PCS and MCS and 43.6 [9.4] for FACIT-F), which demonstrates the significant impact that CAPS has on patients' well-being. The scores improved rapidly after one single dose of canakinumab and approached levels of the general population after 8 weeks (SF-36® PCS, 48.4 [8.7] and MCS, 52.8 [7.7]; FACIT-F, 41.7 [9.3]). This improvement was maintained at one year, as shown by the SF-36® PCS (49.8 [8.6]) and MCS (51.2 [9.9]) and FACIT-F (42.9 [10]) scores. For patients who continued the study beyond one year for up to two years HRQoL scores remained within the range of general population at the final visit. An improvement in functional disability was also observed as shown by HAQ-DI scores, although the level of functional disability in these patients as measured by the HAQ was not marked. Patients who rolled-over from previous studies while maintaining the 8 weeks dosing schedule already had HRQoL scores close to general population levels at baseline and these levels were maintained throughout the study.

Conclusions:

Canakinumab administration improved the HRQoL scores to levels seen in the general population in a large cohort of CAPS patients across all severity phenotypes.

To cite this abstract, please use the following information:
Kone-Paut, I., Lachmann, H. J., Kuemmerle-Deschner, J. B., Hachulla, E., Cartwright, R., Hoyer, J., et al; Canakinumab (ILARIS) Improves Health-Related Quality of Life (HRQoL) in Patients with Cryopyrin-Associated Periodic Fever Syndrome (CAPS): Results of a Phase III, Open-Label Study in a Large Cohort of CAPS Patients. [abstract]. Arthritis Rheum 2010;62 Suppl 10 :1425
DOI: 10.1002/art.29191

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