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Type: Artigo de periódico
Title: Results from a large multinational clinical trial (guardian (TM) 1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy
Author: Lentz, SR
Misgav, M
Ozelo, M
Salek, SZ
Veljkovic, D
Recht, M
Cerqueira, M
Tiede, A
Brand, B
Mancuso, ME
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.
Subject: haemophilia A
recombinant factor VIII
turoctocog alfa
Country: EUA
Editor: Wiley-blackwell
Citation: Haemophilia. Wiley-blackwell, v. 19, n. 5, n. 691, n. 697, 2013.
Rights: fechado
Identifier DOI: 10.1111/hae.12159
Date Issue: 2013
Appears in Collections:Unicamp - Artigos e Outros Documentos

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