Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A: Final results from the guardian 2 extension trial

Steven R. Lentz, Dragana Janic, Kaan Kavakli, Predrag Miljic, Johannes Oldenburg, Margareth C. Ozelo, Elena Santagostino, Takashi Suzuki, Silva Zupancic Šalek, Lars Korsholm, Irina Matytsina, Andreas Tiede

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Abstract

Introduction: Turoctocog alfa is a recombinant factor VIII (FVIII) molecule, approved for treatment and prophylaxis of bleeding in patients with haemophilia A. In the guardian 1 (adolescents/adults) and guardian 3 (children) phase 3 trials, turoctocog alfa demonstrated a favourable efficacy and safety profile. Guardian 1 or 3 completers could enrol in the guardian 2 extension. Final guardian 2 results are reported here. Aim: Investigate long-term safety and efficacy of turoctocog alfa administered for prophylaxis and treatment of bleeds. Methods: In this phase 3b open-label trial, previously treated males of all ages with severe haemophilia A received prophylaxis regimens of turoctocog alfa or on-demand treatment of bleeds. The primary safety endpoint was frequency of FVIII inhibitor development. Efficacy endpoints included annualized bleeding rate (ABR) during prophylaxis, haemostatic response in treatment of bleeds and number of injections required to treat bleeds. Results: Overall, 213 patients were dosed with turoctocog alfa; 207 patients received prophylaxis; 19 received on-demand treatment. No FVIII inhibitors (≥0.6 BU) were reported. For all patients on prophylaxis, overall median ABR was 1.37 bleeds/y; success rate for treatment of bleeds was 90.2%; and 88.2% of bleeds were controlled with 1-2 injections of turoctocog alfa. For the on-demand regimen, overall median ABR was 30.44 bleeds/y; success rate for treatment of bleeds was 96.7%; and 94.9% of bleeds were controlled with 1-2 injections of turoctocog alfa. Conclusion: Extended use of turoctocog alfa is safe and effective for prevention and treatment of bleeding episodes in previously treated patients with haemophilia A across all ages.

Original languageEnglish
Pages (from-to)e391-e394
JournalHaemophilia
Volume24
Issue number6
DOIs
Publication statusPublished - 2018

Fingerprint

Hemophilia A
Hemorrhage
Safety
Factor VIII
Therapeutics
Injections
recombinant factor VIII N8
Hemostatics

Keywords

  • annualized bleeding rate
  • haemophilia A
  • prophylaxis
  • recombinant factor VIII
  • turoctocog alfa

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A : Final results from the guardian 2 extension trial. / Lentz, Steven R.; Janic, Dragana; Kavakli, Kaan; Miljic, Predrag; Oldenburg, Johannes; C. Ozelo, Margareth; Santagostino, Elena; Suzuki, Takashi; Zupancic Šalek, Silva; Korsholm, Lars; Matytsina, Irina; Tiede, Andreas.

In: Haemophilia, Vol. 24, No. 6, 2018, p. e391-e394.

Research output: Contribution to journalArticle

Lentz, SR, Janic, D, Kavakli, K, Miljic, P, Oldenburg, J, C. Ozelo, M, Santagostino, E, Suzuki, T, Zupancic Šalek, S, Korsholm, L, Matytsina, I & Tiede, A 2018, 'Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A: Final results from the guardian 2 extension trial', Haemophilia, vol. 24, no. 6, pp. e391-e394. https://doi.org/10.1111/hae.13617
Lentz, Steven R. ; Janic, Dragana ; Kavakli, Kaan ; Miljic, Predrag ; Oldenburg, Johannes ; C. Ozelo, Margareth ; Santagostino, Elena ; Suzuki, Takashi ; Zupancic Šalek, Silva ; Korsholm, Lars ; Matytsina, Irina ; Tiede, Andreas. / Long-term safety and efficacy of turoctocog alfa in prophylaxis and treatment of bleeding episodes in severe haemophilia A : Final results from the guardian 2 extension trial. In: Haemophilia. 2018 ; Vol. 24, No. 6. pp. e391-e394.
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abstract = "Introduction: Turoctocog alfa is a recombinant factor VIII (FVIII) molecule, approved for treatment and prophylaxis of bleeding in patients with haemophilia A. In the guardian 1 (adolescents/adults) and guardian 3 (children) phase 3 trials, turoctocog alfa demonstrated a favourable efficacy and safety profile. Guardian 1 or 3 completers could enrol in the guardian 2 extension. Final guardian 2 results are reported here. Aim: Investigate long-term safety and efficacy of turoctocog alfa administered for prophylaxis and treatment of bleeds. Methods: In this phase 3b open-label trial, previously treated males of all ages with severe haemophilia A received prophylaxis regimens of turoctocog alfa or on-demand treatment of bleeds. The primary safety endpoint was frequency of FVIII inhibitor development. Efficacy endpoints included annualized bleeding rate (ABR) during prophylaxis, haemostatic response in treatment of bleeds and number of injections required to treat bleeds. Results: Overall, 213 patients were dosed with turoctocog alfa; 207 patients received prophylaxis; 19 received on-demand treatment. No FVIII inhibitors (≥0.6 BU) were reported. For all patients on prophylaxis, overall median ABR was 1.37 bleeds/y; success rate for treatment of bleeds was 90.2{\%}; and 88.2{\%} of bleeds were controlled with 1-2 injections of turoctocog alfa. For the on-demand regimen, overall median ABR was 30.44 bleeds/y; success rate for treatment of bleeds was 96.7{\%}; and 94.9{\%} of bleeds were controlled with 1-2 injections of turoctocog alfa. Conclusion: Extended use of turoctocog alfa is safe and effective for prevention and treatment of bleeding episodes in previously treated patients with haemophilia A across all ages.",
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AU - Janic, Dragana

AU - Kavakli, Kaan

AU - Miljic, Predrag

AU - Oldenburg, Johannes

AU - C. Ozelo, Margareth

AU - Santagostino, Elena

AU - Suzuki, Takashi

AU - Zupancic Šalek, Silva

AU - Korsholm, Lars

AU - Matytsina, Irina

AU - Tiede, Andreas

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N2 - Introduction: Turoctocog alfa is a recombinant factor VIII (FVIII) molecule, approved for treatment and prophylaxis of bleeding in patients with haemophilia A. In the guardian 1 (adolescents/adults) and guardian 3 (children) phase 3 trials, turoctocog alfa demonstrated a favourable efficacy and safety profile. Guardian 1 or 3 completers could enrol in the guardian 2 extension. Final guardian 2 results are reported here. Aim: Investigate long-term safety and efficacy of turoctocog alfa administered for prophylaxis and treatment of bleeds. Methods: In this phase 3b open-label trial, previously treated males of all ages with severe haemophilia A received prophylaxis regimens of turoctocog alfa or on-demand treatment of bleeds. The primary safety endpoint was frequency of FVIII inhibitor development. Efficacy endpoints included annualized bleeding rate (ABR) during prophylaxis, haemostatic response in treatment of bleeds and number of injections required to treat bleeds. Results: Overall, 213 patients were dosed with turoctocog alfa; 207 patients received prophylaxis; 19 received on-demand treatment. No FVIII inhibitors (≥0.6 BU) were reported. For all patients on prophylaxis, overall median ABR was 1.37 bleeds/y; success rate for treatment of bleeds was 90.2%; and 88.2% of bleeds were controlled with 1-2 injections of turoctocog alfa. For the on-demand regimen, overall median ABR was 30.44 bleeds/y; success rate for treatment of bleeds was 96.7%; and 94.9% of bleeds were controlled with 1-2 injections of turoctocog alfa. Conclusion: Extended use of turoctocog alfa is safe and effective for prevention and treatment of bleeding episodes in previously treated patients with haemophilia A across all ages.

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KW - recombinant factor VIII

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