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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 |
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| Authors: | Isabelle Koné-Paut Helen J Lachmann Jasmin B Kuemmerle-Deschner Eric Hachulla Kieron S Leslie Richard Mouy Alberto Ferreira Karine Lheritier Neha Patel Ralph Preiss Philip N Hawkins |
| Institution: | 1. Centre de Référence des Maladies Autoinflammatoires, H?pital 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-Stra?e 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, H?pital 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 |
| Abstract: | IntroductionTo 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).MethodsIn 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.ResultsRapid 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.ConclusionsCanakinumab, 150 mg, 8-weekly, induced rapid and sustained remission of symptoms in patients with CAPS, accompanied by substantial improvements in HRQoL.Trial registrationClintrials.gov NCT00465985 |
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