Periprocedural management of type 2N von Willebrand disease with efanesoctocog alfa

J Thromb Haemost. 2023 Dec;21(12):3508-3510. doi: 10.1016/j.jtha.2023.09.009. Epub 2023 Sep 20.

Abstract

Type 2 Normandy von Willebrand disease (type 2N VWD) is a rare qualitative defect in von Willebrand factor (VWF) that results in impaired factor VIII (FVIII) binding and consequently reduced FVIII levels. Current perioperative strategies require VWF concentrates to attain durable hemostatic FVIII levels. This case highlights the successful perioperative management of a 78-year-old female with type 2N VWD and coronary artery disease utilizing efanesoctocog alfa, a novel long-acting recombinant FVIII product approved for hemophilia A. By decoupling the FVIII-VWF interaction, efanesoctocog alfa achieves prolonged FVIII circulation independent of VWF. A single administration targeting 90% FVIII levels yielded sustained FVIII elevation without achieving supraphysiologic VWF levels, thus mitigating potential cardiovascular risks. This is the first report of efanesoctocog alfa use in type 2N VWD. Further clinical studies are necessary to corroborate its efficacy and safety for this indication.

Keywords: blood coagulation; factor VIII; hemostasis; von Willebrand disease; von Willebrand factor.

Publication types

  • Case Reports

MeSH terms

  • Aged
  • Factor VIII / metabolism
  • Factor VIII / therapeutic use
  • Female
  • Hemophilia A* / diagnosis
  • Hemophilia A* / drug therapy
  • Hemostasis
  • Hemostatics*
  • Humans
  • von Willebrand Disease, Type 2* / diagnosis
  • von Willebrand Disease, Type 2* / drug therapy
  • von Willebrand Diseases* / drug therapy
  • von Willebrand Factor / metabolism
  • von Willebrand Factor / therapeutic use

Substances

  • von Willebrand Factor
  • Factor VIII
  • Hemostatics