Safety and efficacy of BAY 94-9027, an extended-half-life factor VIII, during surgery in patients with severe hemophilia A

Results of the PROTECT VIII clinical trial

Elena Santagostino, Shadan Lalezari, Mark T. Reding, Jonathan M Ducore, Heng Joo Ng, Lone H. Poulsen, Lisa A. Michaels, Camila C.G. Linardi

Research output: Contribution to journalArticle

Abstract

Introduction: Ensuring hemostasis during invasive procedures is a challenge in patients with severe hemophilia A. This analysis evaluated efficacy and safety of BAY 94-9027, an extended-half-life recombinant factor VIII (FVIII), in the surgical setting. Materials and methods: Patients participating in an open-label BAY 94-9027 clinical trial who underwent major surgery were included in the analysis. Investigator/surgeon assessment of hemostasis during surgery was the primary outcome. In addition, information about FVIII use, FVIII levels during perioperative period, bleeding complications and FVIII inhibitor development were collected. Results: Data were analyzed for 26 major surgeries (orthopedic, n = 21) in 20 patients aged 13–61 years. BAY 94-9027 provided effective hemostasis during all procedures. FVIII levels 6–8 h post preoperative infusion and prior to the first follow-up infusion were in the range expected to maintain protection in the major surgery setting. The median time from preoperative infusion to the first follow-up infusion (the first infusion administered after the preoperative infusion) was 12.33 (3.6–49.9) h. No intraoperative bleeding complications occurred, and no new inhibitors developed following any surgery. Conclusions: The results of the study demonstrate that BAY 94-9027 was efficacious and well tolerated in the treatment of patients undergoing major surgeries. Advantages of BAY 94-9027 include the potential for less frequent infusion and reduced factor consumption, which should simplify the management of patients during major surgery.

Original languageEnglish (US)
Pages (from-to)13-19
Number of pages7
JournalThrombosis research
Volume183
DOIs
StatePublished - Nov 1 2019

Fingerprint

Factor VIII
Hemophilia A
Half-Life
Clinical Trials
Safety
Hemostasis
Hemorrhage
Perioperative Period
Intraoperative Complications
Orthopedics
BAY 94-9027
Research Personnel

Keywords

  • Extended-half-life factor VIII
  • Hemophilia A
  • Recombinant protein
  • Surgery

ASJC Scopus subject areas

  • Hematology

Cite this

Safety and efficacy of BAY 94-9027, an extended-half-life factor VIII, during surgery in patients with severe hemophilia A : Results of the PROTECT VIII clinical trial. / Santagostino, Elena; Lalezari, Shadan; Reding, Mark T.; Ducore, Jonathan M; Ng, Heng Joo; Poulsen, Lone H.; Michaels, Lisa A.; Linardi, Camila C.G.

In: Thrombosis research, Vol. 183, 01.11.2019, p. 13-19.

Research output: Contribution to journalArticle

Santagostino, Elena ; Lalezari, Shadan ; Reding, Mark T. ; Ducore, Jonathan M ; Ng, Heng Joo ; Poulsen, Lone H. ; Michaels, Lisa A. ; Linardi, Camila C.G. / Safety and efficacy of BAY 94-9027, an extended-half-life factor VIII, during surgery in patients with severe hemophilia A : Results of the PROTECT VIII clinical trial. In: Thrombosis research. 2019 ; Vol. 183. pp. 13-19.
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abstract = "Introduction: Ensuring hemostasis during invasive procedures is a challenge in patients with severe hemophilia A. This analysis evaluated efficacy and safety of BAY 94-9027, an extended-half-life recombinant factor VIII (FVIII), in the surgical setting. Materials and methods: Patients participating in an open-label BAY 94-9027 clinical trial who underwent major surgery were included in the analysis. Investigator/surgeon assessment of hemostasis during surgery was the primary outcome. In addition, information about FVIII use, FVIII levels during perioperative period, bleeding complications and FVIII inhibitor development were collected. Results: Data were analyzed for 26 major surgeries (orthopedic, n = 21) in 20 patients aged 13–61 years. BAY 94-9027 provided effective hemostasis during all procedures. FVIII levels 6–8 h post preoperative infusion and prior to the first follow-up infusion were in the range expected to maintain protection in the major surgery setting. The median time from preoperative infusion to the first follow-up infusion (the first infusion administered after the preoperative infusion) was 12.33 (3.6–49.9) h. No intraoperative bleeding complications occurred, and no new inhibitors developed following any surgery. Conclusions: The results of the study demonstrate that BAY 94-9027 was efficacious and well tolerated in the treatment of patients undergoing major surgeries. Advantages of BAY 94-9027 include the potential for less frequent infusion and reduced factor consumption, which should simplify the management of patients during major surgery.",
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AU - Lalezari, Shadan

AU - Reding, Mark T.

AU - Ducore, Jonathan M

AU - Ng, Heng Joo

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AU - Michaels, Lisa A.

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N2 - Introduction: Ensuring hemostasis during invasive procedures is a challenge in patients with severe hemophilia A. This analysis evaluated efficacy and safety of BAY 94-9027, an extended-half-life recombinant factor VIII (FVIII), in the surgical setting. Materials and methods: Patients participating in an open-label BAY 94-9027 clinical trial who underwent major surgery were included in the analysis. Investigator/surgeon assessment of hemostasis during surgery was the primary outcome. In addition, information about FVIII use, FVIII levels during perioperative period, bleeding complications and FVIII inhibitor development were collected. Results: Data were analyzed for 26 major surgeries (orthopedic, n = 21) in 20 patients aged 13–61 years. BAY 94-9027 provided effective hemostasis during all procedures. FVIII levels 6–8 h post preoperative infusion and prior to the first follow-up infusion were in the range expected to maintain protection in the major surgery setting. The median time from preoperative infusion to the first follow-up infusion (the first infusion administered after the preoperative infusion) was 12.33 (3.6–49.9) h. No intraoperative bleeding complications occurred, and no new inhibitors developed following any surgery. Conclusions: The results of the study demonstrate that BAY 94-9027 was efficacious and well tolerated in the treatment of patients undergoing major surgeries. Advantages of BAY 94-9027 include the potential for less frequent infusion and reduced factor consumption, which should simplify the management of patients during major surgery.

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