Efficacy and safety of a VWF/FVIII concentrate (wilate®) in inherited von Willebrand disease patients undergoing surgical procedures

A. Srivastava, M. Serban, S. Werner, B. A. Schwartz, C. M. Kessler, Salam Alkindi, Shashikant Janardan Apte, Giancarlo Castaman, Joan Cox Gill, Andrzej Hellmann, Kaan Kavakli, Peter A. Kouides, Philip Kuriakose, Toshko Lissitchkov, Alice Ma, Johnny Mahlangu, Massimo Morfini, Ellis J. Neufeld, Flora Peyvandi, Mark Reding & 8 others Nidra I. Rodriguez, Cecil Ross, Luminita Rusen, Amy Shapiro, Valentina Uscatescu, Jerzy Windyga, Bulent Zulfikar, the Wonders Study Investigators

Research output: Contribution to journalArticle

7 Citations (Scopus)

Abstract

Introduction: Surgical procedures in von Willebrand disease (VWD) patients may require prophylactic treatment with exogenous von Willebrand factor (VWF) and coagulation factor VIII (FVIII) to prevent excessive bleeding. Wilate® is a plasma-derived, double virus-inactivated, highly purified, freeze-dried VWF/FVIII concentrate, containing both factors in a physiological activity ratio of 1:1. Aim: To investigate the efficacy and safety of wilate® in maintaining haemostasis in VWD patients undergoing surgical procedures. Methods: This prospective, open-label multinational clinical study documents 28 individuals who underwent 30 surgical procedures managed with wilate®. Twenty-one patients had VWD Type 3, and 21 surgeries were major. Efficacy was assessed intra- and postoperatively by the surgeon and investigator, respectively, and adjudicated by an Independent Data Monitoring Committee, using an objective scale based on blood loss, transfusion requirements and postoperative bleeding and oozing. Treatment success (primary endpoint) was determined using a composite assessment algorithm and was formally assessed. Results: Surgical prophylaxis with wilate® was successful in 29 of 30 procedures. The overall rate of success was 96.7% (98.75% CI: 0.784, 1.000). All 21 surgeries in patients with VWD Type 3 were managed successfully. There was no accumulation of VWF or FVIII after multiple dosing, and no thromboembolic events or inhibitors to VWF or FVIII were observed. Conclusions: Wilate® demonstrated effective prevention and treatment of bleeding in inherited VWD patients undergoing surgery, with no clinically significant safety concerns.

Original languageEnglish
Pages (from-to)264-272
Number of pages9
JournalHaemophilia
Volume23
Issue number2
DOIs
Publication statusPublished - Mar 1 2017

Fingerprint

von Willebrand Diseases
Type 3 Von Willebrand's Disease
Safety
Hemorrhage
Clinical Trials Data Monitoring Committees
Factor VIII
von Willebrand Factor
Hemostasis
Blood Transfusion
Therapeutics
Research Personnel
factor VIII, von Willebrand factor drug combination
Viruses

Keywords

  • coagulation factor VIII
  • surgery
  • von Willebrand disease
  • von Willebrand factor
  • wilate

ASJC Scopus subject areas

  • Hematology
  • Genetics(clinical)

Cite this

Srivastava, A., Serban, M., Werner, S., Schwartz, B. A., Kessler, C. M., Alkindi, S., ... the Wonders Study Investigators (2017). Efficacy and safety of a VWF/FVIII concentrate (wilate®) in inherited von Willebrand disease patients undergoing surgical procedures. Haemophilia, 23(2), 264-272. https://doi.org/10.1111/hae.13106

Efficacy and safety of a VWF/FVIII concentrate (wilate®) in inherited von Willebrand disease patients undergoing surgical procedures. / Srivastava, A.; Serban, M.; Werner, S.; Schwartz, B. A.; Kessler, C. M.; Alkindi, Salam; Apte, Shashikant Janardan; Castaman, Giancarlo; Gill, Joan Cox; Hellmann, Andrzej; Kavakli, Kaan; Kouides, Peter A.; Kuriakose, Philip; Lissitchkov, Toshko; Ma, Alice; Mahlangu, Johnny; Morfini, Massimo; Neufeld, Ellis J.; Peyvandi, Flora; Reding, Mark; Rodriguez, Nidra I.; Ross, Cecil; Rusen, Luminita; Shapiro, Amy; Uscatescu, Valentina; Windyga, Jerzy; Zulfikar, Bulent; the Wonders Study Investigators.

In: Haemophilia, Vol. 23, No. 2, 01.03.2017, p. 264-272.

Research output: Contribution to journalArticle

Srivastava, A, Serban, M, Werner, S, Schwartz, BA, Kessler, CM, Alkindi, S, Apte, SJ, Castaman, G, Gill, JC, Hellmann, A, Kavakli, K, Kouides, PA, Kuriakose, P, Lissitchkov, T, Ma, A, Mahlangu, J, Morfini, M, Neufeld, EJ, Peyvandi, F, Reding, M, Rodriguez, NI, Ross, C, Rusen, L, Shapiro, A, Uscatescu, V, Windyga, J, Zulfikar, B & the Wonders Study Investigators 2017, 'Efficacy and safety of a VWF/FVIII concentrate (wilate®) in inherited von Willebrand disease patients undergoing surgical procedures', Haemophilia, vol. 23, no. 2, pp. 264-272. https://doi.org/10.1111/hae.13106
Srivastava, A. ; Serban, M. ; Werner, S. ; Schwartz, B. A. ; Kessler, C. M. ; Alkindi, Salam ; Apte, Shashikant Janardan ; Castaman, Giancarlo ; Gill, Joan Cox ; Hellmann, Andrzej ; Kavakli, Kaan ; Kouides, Peter A. ; Kuriakose, Philip ; Lissitchkov, Toshko ; Ma, Alice ; Mahlangu, Johnny ; Morfini, Massimo ; Neufeld, Ellis J. ; Peyvandi, Flora ; Reding, Mark ; Rodriguez, Nidra I. ; Ross, Cecil ; Rusen, Luminita ; Shapiro, Amy ; Uscatescu, Valentina ; Windyga, Jerzy ; Zulfikar, Bulent ; the Wonders Study Investigators. / Efficacy and safety of a VWF/FVIII concentrate (wilate®) in inherited von Willebrand disease patients undergoing surgical procedures. In: Haemophilia. 2017 ; Vol. 23, No. 2. pp. 264-272.
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abstract = "Introduction: Surgical procedures in von Willebrand disease (VWD) patients may require prophylactic treatment with exogenous von Willebrand factor (VWF) and coagulation factor VIII (FVIII) to prevent excessive bleeding. Wilate{\circledR} is a plasma-derived, double virus-inactivated, highly purified, freeze-dried VWF/FVIII concentrate, containing both factors in a physiological activity ratio of 1:1. Aim: To investigate the efficacy and safety of wilate{\circledR} in maintaining haemostasis in VWD patients undergoing surgical procedures. Methods: This prospective, open-label multinational clinical study documents 28 individuals who underwent 30 surgical procedures managed with wilate{\circledR}. Twenty-one patients had VWD Type 3, and 21 surgeries were major. Efficacy was assessed intra- and postoperatively by the surgeon and investigator, respectively, and adjudicated by an Independent Data Monitoring Committee, using an objective scale based on blood loss, transfusion requirements and postoperative bleeding and oozing. Treatment success (primary endpoint) was determined using a composite assessment algorithm and was formally assessed. Results: Surgical prophylaxis with wilate{\circledR} was successful in 29 of 30 procedures. The overall rate of success was 96.7{\%} (98.75{\%} CI: 0.784, 1.000). All 21 surgeries in patients with VWD Type 3 were managed successfully. There was no accumulation of VWF or FVIII after multiple dosing, and no thromboembolic events or inhibitors to VWF or FVIII were observed. Conclusions: Wilate{\circledR} demonstrated effective prevention and treatment of bleeding in inherited VWD patients undergoing surgery, with no clinically significant safety concerns.",
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T1 - Efficacy and safety of a VWF/FVIII concentrate (wilate®) in inherited von Willebrand disease patients undergoing surgical procedures

AU - Srivastava, A.

AU - Serban, M.

AU - Werner, S.

AU - Schwartz, B. A.

AU - Kessler, C. M.

AU - Alkindi, Salam

AU - Apte, Shashikant Janardan

AU - Castaman, Giancarlo

AU - Gill, Joan Cox

AU - Hellmann, Andrzej

AU - Kavakli, Kaan

AU - Kouides, Peter A.

AU - Kuriakose, Philip

AU - Lissitchkov, Toshko

AU - Ma, Alice

AU - Mahlangu, Johnny

AU - Morfini, Massimo

AU - Neufeld, Ellis J.

AU - Peyvandi, Flora

AU - Reding, Mark

AU - Rodriguez, Nidra I.

AU - Ross, Cecil

AU - Rusen, Luminita

AU - Shapiro, Amy

AU - Uscatescu, Valentina

AU - Windyga, Jerzy

AU - Zulfikar, Bulent

AU - the Wonders Study Investigators

PY - 2017/3/1

Y1 - 2017/3/1

N2 - Introduction: Surgical procedures in von Willebrand disease (VWD) patients may require prophylactic treatment with exogenous von Willebrand factor (VWF) and coagulation factor VIII (FVIII) to prevent excessive bleeding. Wilate® is a plasma-derived, double virus-inactivated, highly purified, freeze-dried VWF/FVIII concentrate, containing both factors in a physiological activity ratio of 1:1. Aim: To investigate the efficacy and safety of wilate® in maintaining haemostasis in VWD patients undergoing surgical procedures. Methods: This prospective, open-label multinational clinical study documents 28 individuals who underwent 30 surgical procedures managed with wilate®. Twenty-one patients had VWD Type 3, and 21 surgeries were major. Efficacy was assessed intra- and postoperatively by the surgeon and investigator, respectively, and adjudicated by an Independent Data Monitoring Committee, using an objective scale based on blood loss, transfusion requirements and postoperative bleeding and oozing. Treatment success (primary endpoint) was determined using a composite assessment algorithm and was formally assessed. Results: Surgical prophylaxis with wilate® was successful in 29 of 30 procedures. The overall rate of success was 96.7% (98.75% CI: 0.784, 1.000). All 21 surgeries in patients with VWD Type 3 were managed successfully. There was no accumulation of VWF or FVIII after multiple dosing, and no thromboembolic events or inhibitors to VWF or FVIII were observed. Conclusions: Wilate® demonstrated effective prevention and treatment of bleeding in inherited VWD patients undergoing surgery, with no clinically significant safety concerns.

AB - Introduction: Surgical procedures in von Willebrand disease (VWD) patients may require prophylactic treatment with exogenous von Willebrand factor (VWF) and coagulation factor VIII (FVIII) to prevent excessive bleeding. Wilate® is a plasma-derived, double virus-inactivated, highly purified, freeze-dried VWF/FVIII concentrate, containing both factors in a physiological activity ratio of 1:1. Aim: To investigate the efficacy and safety of wilate® in maintaining haemostasis in VWD patients undergoing surgical procedures. Methods: This prospective, open-label multinational clinical study documents 28 individuals who underwent 30 surgical procedures managed with wilate®. Twenty-one patients had VWD Type 3, and 21 surgeries were major. Efficacy was assessed intra- and postoperatively by the surgeon and investigator, respectively, and adjudicated by an Independent Data Monitoring Committee, using an objective scale based on blood loss, transfusion requirements and postoperative bleeding and oozing. Treatment success (primary endpoint) was determined using a composite assessment algorithm and was formally assessed. Results: Surgical prophylaxis with wilate® was successful in 29 of 30 procedures. The overall rate of success was 96.7% (98.75% CI: 0.784, 1.000). All 21 surgeries in patients with VWD Type 3 were managed successfully. There was no accumulation of VWF or FVIII after multiple dosing, and no thromboembolic events or inhibitors to VWF or FVIII were observed. Conclusions: Wilate® demonstrated effective prevention and treatment of bleeding in inherited VWD patients undergoing surgery, with no clinically significant safety concerns.

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