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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 and Berntorp, Erik LU (2019) In Blood Reviews 38.
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... (More)

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.

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author
; ; ; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Acute bleeding, Multimer, Plasma-derived, Prophylaxis in VWD, Recombinant, von Willebrand factor
in
Blood Reviews
volume
38
article number
100572
publisher
Churchill Livingstone
external identifiers
  • pmid:31229334
  • scopus:85067342769
ISSN
0268-960X
DOI
10.1016/j.blre.2019.04.001
language
English
LU publication?
yes
id
64ff1a0f-6afb-48c6-82cf-58ed1db6008c
date added to LUP
2019-07-02 16:45:37
date last changed
2024-05-01 15:38:21
@article{64ff1a0f-6afb-48c6-82cf-58ed1db6008c,
  abstract     = {{<p>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 &gt;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.</p>}},
  author       = {{Peyvandi, Flora and Kouides, Peter and Turecek, Peter L. and Dow, Edward and Berntorp, Erik}},
  issn         = {{0268-960X}},
  keywords     = {{Acute bleeding; Multimer; Plasma-derived; Prophylaxis in VWD; Recombinant; von Willebrand factor}},
  language     = {{eng}},
  publisher    = {{Churchill Livingstone}},
  series       = {{Blood Reviews}},
  title        = {{Evolution of replacement therapy for von Willebrand disease : From plasma fraction to recombinant von Willebrand factor}},
  url          = {{http://dx.doi.org/10.1016/j.blre.2019.04.001}},
  doi          = {{10.1016/j.blre.2019.04.001}},
  volume       = {{38}},
  year         = {{2019}},
}