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Open Access Research article

Sustained remission of symptoms and improved health-related quality of life in patients with cryopyrin-associated periodic syndrome treated with canakinumab: results of a double-blind placebo-controlled randomized withdrawal study

Isabelle Koné-Paut1*, Helen J Lachmann2, Jasmin B Kuemmerle-Deschner3, Eric Hachulla4, Kieron S Leslie5, Richard Mouy6, Alberto Ferreira7, Karine Lheritier7, Neha Patel8, Ralph Preiss8, Philip N Hawkins2 and on behalf of the Canakinumab in CAPS Study Group

Author Affiliations

1 Centre de Référence des Maladies Autoinflammatoires, Hôpital Kremlin Bicetre, Paris University of Medicine, Le Kremlin Bicetre, Paris, France

2 UCL Medical School, Gower Street, London, WC1E 6BT, UK

3 Division of Pediatric Rheumatology, Department of Pediatrics, University Hospital Tübingen, Hoppe-Seyler-Straße 1, 72076 Tuebingen, Germany

4 Department of Internal Medicine, Claude Huriez Hospital, University of Lille, Lille Cedex, France

5 University of California at San Francisco, San Francisco, CA 94143, USA

6 Unité de Rhumatologie Pédiatrique, Hôpital Necker-Enfants Malades, 149 rue de Sèvres, 75015 Paris, France

7 Novartis Pharma AG, CH-4002, Basel, Switzerland

8 Novartis Pharmaceuticals Corporation, One Health Plaza, East Hanover, NJ 07936-1080, USA

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Arthritis Research & Therapy 2011, 13:R202  doi:10.1186/ar3535

Published: 9 December 2011

Abstract

Introduction

To assess the effect of canakinumab, a fully human anti-interleukin-1β antibody, on symptoms and health-related quality of life (HRQoL) in patients with cryopyrin-associated periodic syndrome (CAPS).

Methods

In this 48-week, phase 3 study, patients with CAPS received canakinumab 150 mg subcutaneously at 8-week intervals. All patients (n = 35) received canakinumab during weeks 1 through 8; weeks 9 through 24 constituted a double-blind placebo-controlled withdrawal phase, and weeks 24 through 48 constituted an open-label phase in which all patients received canakinumab. Patient and physician assessments of symptoms, levels of inflammatory markers, and HRQoL were performed.

Results

Rapid symptom remission was achieved, with 89% of patients having no or minimal disease activity on day 8. Responses were sustained in patients receiving 8-weekly canakinumab. Responses were lost during the placebo-controlled phase in the placebo group and were regained on resuming canakinumab therapy in the open-label phase. Clinical responses were accompanied by decreases in serum levels of C-reactive protein, serum amyloid A protein, and interleukin-6. HRQoL scores at baseline were considerably below those of the general population. Improvements in all 36-item Short-Form Health Survey (SF-36) domain scores were evident by day 8. Scores approached or exceeded those of the general U.S. population by week 8 and remained stable during canakinumab therapy. Improvements in bodily pain and role-physical were particularly marked, increasing by more than 25 points from baseline to week 8. Therapy was generally well tolerated.

Conclusions

Canakinumab, 150 mg, 8-weekly, induced rapid and sustained remission of symptoms in patients with CAPS, accompanied by substantial improvements in HRQoL.

Clintrials.gov NCT00465985