Alloantibodies in von Willebrand disease

Paula D. James, David Lillicrap, Pier M. Mannucci

Research output: Contribution to journalArticle

44 Citations (Scopus)

Abstract

The development of alloantibodies against von Willebrand factor (VWF) represents a rare but serious complication of treatment of von Willebrand disease (VWD), occurring in ∼5% to 10% of type 3 VWD patients. Affected patients can present with a range of symptoms, including lack or loss of hemostatic response to infused VWF concentrates up to anaphylactic reactions in rare cases. It is classically reported in multitransfused patients and occurs most frequently in patients with partial or complete VWF gene deletions. A positive family history of anti-VWF antibodies also appears to be a risk factor. There is a lack of standardization of laboratory methods for antibody identification and characterization. Issues of variability in laboratory approaches as well as the rarity of the complication act as a barrier to future studies. Recombinant factor VIII as well as bypassing agents and immune tolerance have been reported as effective treatments; however, aside from case reports, little exists in the literature to guide management. The imminent clinical availability of recombinant VWF has prompted a resurgence of interest in this area. Additional study is warranted to address the deficiencies in our understanding of this treatment complication.

Original languageEnglish
Pages (from-to)636-640
Number of pages5
JournalBlood
Volume122
Issue number5
DOIs
Publication statusPublished - Aug 1 2013

Fingerprint

Isoantibodies
von Willebrand Diseases
von Willebrand Factor
Type 3 Von Willebrand's Disease
Immune Tolerance
Antibodies
Gene Deletion
Factor VIII
Anaphylaxis
Hemostatics
Standardization
Therapeutics
Genes
Availability

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Cite this

Alloantibodies in von Willebrand disease. / James, Paula D.; Lillicrap, David; Mannucci, Pier M.

In: Blood, Vol. 122, No. 5, 01.08.2013, p. 636-640.

Research output: Contribution to journalArticle

James, Paula D. ; Lillicrap, David ; Mannucci, Pier M. / Alloantibodies in von Willebrand disease. In: Blood. 2013 ; Vol. 122, No. 5. pp. 636-640.
@article{e1b3645f13914b0e96a28575c49befc6,
title = "Alloantibodies in von Willebrand disease",
abstract = "The development of alloantibodies against von Willebrand factor (VWF) represents a rare but serious complication of treatment of von Willebrand disease (VWD), occurring in ∼5{\%} to 10{\%} of type 3 VWD patients. Affected patients can present with a range of symptoms, including lack or loss of hemostatic response to infused VWF concentrates up to anaphylactic reactions in rare cases. It is classically reported in multitransfused patients and occurs most frequently in patients with partial or complete VWF gene deletions. A positive family history of anti-VWF antibodies also appears to be a risk factor. There is a lack of standardization of laboratory methods for antibody identification and characterization. Issues of variability in laboratory approaches as well as the rarity of the complication act as a barrier to future studies. Recombinant factor VIII as well as bypassing agents and immune tolerance have been reported as effective treatments; however, aside from case reports, little exists in the literature to guide management. The imminent clinical availability of recombinant VWF has prompted a resurgence of interest in this area. Additional study is warranted to address the deficiencies in our understanding of this treatment complication.",
author = "James, {Paula D.} and David Lillicrap and Mannucci, {Pier M.}",
year = "2013",
month = "8",
day = "1",
doi = "10.1182/blood-2012-10-462085",
language = "English",
volume = "122",
pages = "636--640",
journal = "Blood",
issn = "0006-4971",
publisher = "American Society of Hematology",
number = "5",

}

TY - JOUR

T1 - Alloantibodies in von Willebrand disease

AU - James, Paula D.

AU - Lillicrap, David

AU - Mannucci, Pier M.

PY - 2013/8/1

Y1 - 2013/8/1

N2 - The development of alloantibodies against von Willebrand factor (VWF) represents a rare but serious complication of treatment of von Willebrand disease (VWD), occurring in ∼5% to 10% of type 3 VWD patients. Affected patients can present with a range of symptoms, including lack or loss of hemostatic response to infused VWF concentrates up to anaphylactic reactions in rare cases. It is classically reported in multitransfused patients and occurs most frequently in patients with partial or complete VWF gene deletions. A positive family history of anti-VWF antibodies also appears to be a risk factor. There is a lack of standardization of laboratory methods for antibody identification and characterization. Issues of variability in laboratory approaches as well as the rarity of the complication act as a barrier to future studies. Recombinant factor VIII as well as bypassing agents and immune tolerance have been reported as effective treatments; however, aside from case reports, little exists in the literature to guide management. The imminent clinical availability of recombinant VWF has prompted a resurgence of interest in this area. Additional study is warranted to address the deficiencies in our understanding of this treatment complication.

AB - The development of alloantibodies against von Willebrand factor (VWF) represents a rare but serious complication of treatment of von Willebrand disease (VWD), occurring in ∼5% to 10% of type 3 VWD patients. Affected patients can present with a range of symptoms, including lack or loss of hemostatic response to infused VWF concentrates up to anaphylactic reactions in rare cases. It is classically reported in multitransfused patients and occurs most frequently in patients with partial or complete VWF gene deletions. A positive family history of anti-VWF antibodies also appears to be a risk factor. There is a lack of standardization of laboratory methods for antibody identification and characterization. Issues of variability in laboratory approaches as well as the rarity of the complication act as a barrier to future studies. Recombinant factor VIII as well as bypassing agents and immune tolerance have been reported as effective treatments; however, aside from case reports, little exists in the literature to guide management. The imminent clinical availability of recombinant VWF has prompted a resurgence of interest in this area. Additional study is warranted to address the deficiencies in our understanding of this treatment complication.

UR - http://www.scopus.com/inward/record.url?scp=84886395616&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84886395616&partnerID=8YFLogxK

U2 - 10.1182/blood-2012-10-462085

DO - 10.1182/blood-2012-10-462085

M3 - Article

C2 - 23297130

AN - SCOPUS:84886395616

VL - 122

SP - 636

EP - 640

JO - Blood

JF - Blood

SN - 0006-4971

IS - 5

ER -