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

S. R. Lentz, M. Misgav, M. Ozelo, S. Z. Šalek, D. Veljkovic, Michael Recht, M. Cerqueira, A. Tiede, B. Brand, M. E. Mancuso, S. Seremetis, A. Lindblom, U. Martinowitz

Research output: Contribution to journalArticle

52 Citations (Scopus)

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.

Original languageEnglish (US)
Pages (from-to)691-697
Number of pages7
JournalHaemophilia
Volume19
Issue number5
DOIs
StatePublished - Sep 2013

Fingerprint

Hemophilia A
Clinical Trials
Safety
Hemorrhage
Therapeutics
Factor VIII
Nasopharyngitis
recombinant factor VIII N8
Hemostatics
Headache

Keywords

  • Haemophilia A
  • Prophylaxis
  • Recombinant factor VIII
  • Turoctocog alfa

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

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. / Lentz, S. R.; Misgav, M.; Ozelo, M.; Šalek, S. Z.; Veljkovic, D.; Recht, Michael; Cerqueira, M.; Tiede, A.; Brand, B.; Mancuso, M. E.; Seremetis, S.; Lindblom, A.; Martinowitz, U.

In: Haemophilia, Vol. 19, No. 5, 09.2013, p. 691-697.

Research output: Contribution to journalArticle

Lentz, SR, Misgav, M, Ozelo, M, Šalek, SZ, Veljkovic, D, Recht, M, Cerqueira, M, Tiede, A, Brand, B, Mancuso, ME, Seremetis, S, Lindblom, A & Martinowitz, U 2013, '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', Haemophilia, vol. 19, no. 5, pp. 691-697. https://doi.org/10.1111/hae.12159
Lentz, S. R. ; Misgav, M. ; Ozelo, M. ; Šalek, S. Z. ; Veljkovic, D. ; Recht, Michael ; Cerqueira, M. ; Tiede, A. ; Brand, B. ; Mancuso, M. E. ; Seremetis, S. ; Lindblom, A. ; Martinowitz, U. / 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. In: Haemophilia. 2013 ; Vol. 19, No. 5. pp. 691-697.
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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.",
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