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Type: Artigo de periódico
Title: Results From A Large Multinational Clinical Trial (guardian™1) Using Prophylactic Treatment With Turoctocog Alfa In Adolescent And Adult Patients With Severe Haemophilia A: Safety And Efficacy
Author: Lentz S.R.
Misgav M.
Ozelo M.
Salek S.Z.
Veljkovic D.
Recht M.
Cerqueira M.
Tiede A.
Brand B.
Mancuso M.E.
Seremetis S.
Lindblom A.
Martinowitz U.
Abstract: Recombinant factor VIII (rFVIII) products provide a safe and efficacious replacement therapy for prophylaxis and treatment of bleeding episodes in patients with severe haemophilia A. This multinational, open-label, non-controlled trial investigated the safety and efficacy of turoctocog alfa, a new rFVIII product. The primary objective was to evaluate safety. A total of 150 patients (24 adolescents and 126 adults) with severe haemophilia A (FVIII activity ≤1%), with at least 150 exposure days (EDs) to any FVIII product and no history of inhibitors were enrolled, and 146 patients (97%) completed the trial. All patients received prophylaxis with turoctocog alfa for approximately 6 months and had a mean of 85 EDs during the trial. None of the patients developed FVIII inhibitors, there were no indications of early FVIII inhibitor development and no safety concerns were identified. A total of 225 adverse events were reported in 100 (67%) patients, with the most common being events associated with dosing procedures, headaches, and nasopharyngitis. A total of 499 bleeding episodes were reported during the trial, the majority (89%) were controlled with 1-2 infusions of turoctocog alfa. Based on patient reports, the success rate (defined as 'excellent' or 'good' haemostatic response) for treatment of bleeding episodes was 81%. The overall median annualized bleeding rate was 3.7 (interquartile range: 8.7) bleeds/patient/year. In conclusion, turoctocog alfa provides a new, safe and effective alternative for prophylaxis and treatment of bleeding episodes in patients with haemophilia A. © 2013 John Wiley & Sons Ltd.
Rights: fechado
Identifier DOI: 10.1111/hae.12159
Date Issue: 2013
Appears in Collections:Unicamp - Artigos e Outros Documentos

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