Evolution of replacement therapy for von Willebrand disease: From plasma fraction to recombinant von Willebrand factor

Flora Peyvandi, Peter Kouides, Peter L. Turecek, Edward Dow, Erik Berntorp

Research output: Contribution to journalReview article

1 Citation (Scopus)

Abstract

The diagnosis and treatment of von Willebrand disease (VWD) are challenging, in part because patients exhibit a wide range of bleeding patterns and manifestations (e.g. epistaxis, gingival bleeding, heavy menstrual bleeding, gastrointestinal bleeds, postoperative bleeding, hemarthroses) and in part because many tests are required to make an accurate diagnosis. Factor replacement therapies for VWD are the mainstay of treatment for patients who do not respond to desmopressin. They have gradually evolved from crude preparations of plasma proteins to plasma-derived concentrates containing both von Willebrand factor (VWF) and factor VIII (FVIII). However, varying amounts and quality of VWF and varying content of FVIII have contributed to the lack of a standardized approach to replacement therapy. More recently, the treatment of VWD has undergone a slow yet significant change from plasma-derived VWF/FVIII concentrates with VWF:ristocetin cofactor (RCo)/FVIII ratios ≤1, to those with VWF:RCo/FVIII ratios >10, to a recombinant VWF. This article reviews the evolution of factor replacement therapy for patients with VWD that has occurred over the last several decades. The availability of a greater variety of factor replacement therapies poses a challenge in terms of a standard algorithm of care but may help overcome the limitations of earlier treatments and allow treatment personalization according to individual patient needs.

Original languageEnglish
Article number100572
JournalBlood Reviews
Volume38
DOIs
Publication statusPublished - Jan 1 2019

Fingerprint

von Willebrand Diseases
von Willebrand Factor
Factor VIII
Hemorrhage
Therapeutics
Hemarthrosis
Deamino Arginine Vasopressin
Epistaxis
Standard of Care
Blood Proteins

Keywords

  • Acute bleeding
  • Multimer
  • Plasma-derived
  • Prophylaxis in VWD
  • Recombinant
  • von Willebrand factor

ASJC Scopus subject areas

  • Hematology
  • Oncology

Cite this

Evolution of replacement therapy for von Willebrand disease : From plasma fraction to recombinant von Willebrand factor. / Peyvandi, Flora; Kouides, Peter; Turecek, Peter L.; Dow, Edward; Berntorp, Erik.

In: Blood Reviews, Vol. 38, 100572, 01.01.2019.

Research output: Contribution to journalReview article

@article{8a55b5d32f0742808b3210caa1e95ea6,
title = "Evolution of replacement therapy for von Willebrand disease: From plasma fraction to recombinant von Willebrand factor",
abstract = "The diagnosis and treatment of von Willebrand disease (VWD) are challenging, in part because patients exhibit a wide range of bleeding patterns and manifestations (e.g. epistaxis, gingival bleeding, heavy menstrual bleeding, gastrointestinal bleeds, postoperative bleeding, hemarthroses) and in part because many tests are required to make an accurate diagnosis. Factor replacement therapies for VWD are the mainstay of treatment for patients who do not respond to desmopressin. They have gradually evolved from crude preparations of plasma proteins to plasma-derived concentrates containing both von Willebrand factor (VWF) and factor VIII (FVIII). However, varying amounts and quality of VWF and varying content of FVIII have contributed to the lack of a standardized approach to replacement therapy. More recently, the treatment of VWD has undergone a slow yet significant change from plasma-derived VWF/FVIII concentrates with VWF:ristocetin cofactor (RCo)/FVIII ratios ≤1, to those with VWF:RCo/FVIII ratios >10, to a recombinant VWF. This article reviews the evolution of factor replacement therapy for patients with VWD that has occurred over the last several decades. The availability of a greater variety of factor replacement therapies poses a challenge in terms of a standard algorithm of care but may help overcome the limitations of earlier treatments and allow treatment personalization according to individual patient needs.",
keywords = "Acute bleeding, Multimer, Plasma-derived, Prophylaxis in VWD, Recombinant, von Willebrand factor",
author = "Flora Peyvandi and Peter Kouides and Turecek, {Peter L.} and Edward Dow and Erik Berntorp",
year = "2019",
month = "1",
day = "1",
doi = "10.1016/j.blre.2019.04.001",
language = "English",
volume = "38",
journal = "Blood Reviews",
issn = "0268-960X",
publisher = "Churchill Livingstone",

}

TY - JOUR

T1 - Evolution of replacement therapy for von Willebrand disease

T2 - From plasma fraction to recombinant von Willebrand factor

AU - Peyvandi, Flora

AU - Kouides, Peter

AU - Turecek, Peter L.

AU - Dow, Edward

AU - Berntorp, Erik

PY - 2019/1/1

Y1 - 2019/1/1

N2 - The diagnosis and treatment of von Willebrand disease (VWD) are challenging, in part because patients exhibit a wide range of bleeding patterns and manifestations (e.g. epistaxis, gingival bleeding, heavy menstrual bleeding, gastrointestinal bleeds, postoperative bleeding, hemarthroses) and in part because many tests are required to make an accurate diagnosis. Factor replacement therapies for VWD are the mainstay of treatment for patients who do not respond to desmopressin. They have gradually evolved from crude preparations of plasma proteins to plasma-derived concentrates containing both von Willebrand factor (VWF) and factor VIII (FVIII). However, varying amounts and quality of VWF and varying content of FVIII have contributed to the lack of a standardized approach to replacement therapy. More recently, the treatment of VWD has undergone a slow yet significant change from plasma-derived VWF/FVIII concentrates with VWF:ristocetin cofactor (RCo)/FVIII ratios ≤1, to those with VWF:RCo/FVIII ratios >10, to a recombinant VWF. This article reviews the evolution of factor replacement therapy for patients with VWD that has occurred over the last several decades. The availability of a greater variety of factor replacement therapies poses a challenge in terms of a standard algorithm of care but may help overcome the limitations of earlier treatments and allow treatment personalization according to individual patient needs.

AB - The diagnosis and treatment of von Willebrand disease (VWD) are challenging, in part because patients exhibit a wide range of bleeding patterns and manifestations (e.g. epistaxis, gingival bleeding, heavy menstrual bleeding, gastrointestinal bleeds, postoperative bleeding, hemarthroses) and in part because many tests are required to make an accurate diagnosis. Factor replacement therapies for VWD are the mainstay of treatment for patients who do not respond to desmopressin. They have gradually evolved from crude preparations of plasma proteins to plasma-derived concentrates containing both von Willebrand factor (VWF) and factor VIII (FVIII). However, varying amounts and quality of VWF and varying content of FVIII have contributed to the lack of a standardized approach to replacement therapy. More recently, the treatment of VWD has undergone a slow yet significant change from plasma-derived VWF/FVIII concentrates with VWF:ristocetin cofactor (RCo)/FVIII ratios ≤1, to those with VWF:RCo/FVIII ratios >10, to a recombinant VWF. This article reviews the evolution of factor replacement therapy for patients with VWD that has occurred over the last several decades. The availability of a greater variety of factor replacement therapies poses a challenge in terms of a standard algorithm of care but may help overcome the limitations of earlier treatments and allow treatment personalization according to individual patient needs.

KW - Acute bleeding

KW - Multimer

KW - Plasma-derived

KW - Prophylaxis in VWD

KW - Recombinant

KW - von Willebrand factor

UR - http://www.scopus.com/inward/record.url?scp=85067342769&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85067342769&partnerID=8YFLogxK

U2 - 10.1016/j.blre.2019.04.001

DO - 10.1016/j.blre.2019.04.001

M3 - Review article

AN - SCOPUS:85067342769

VL - 38

JO - Blood Reviews

JF - Blood Reviews

SN - 0268-960X

M1 - 100572

ER -