Abstract
Background. von Willebrand disease (VWD) is the most common hereditary bleeding disorder affecting both males and females. It arises from quantitative or qualitative defects of von Willebrand factor (VWF) and causes bleeding of mucous membranes and soft tissues. The aim of treatment is to correct the dual defect of haemostasis caused by the abnormal/reduced VWF and the concomitant deficiency of factor VIII (FVIII). Material and methods. This document contains evidence-based recommendations for the management of VWD compiled by AICE (the Italian Association of Haemophilia Centres). All the evidence supporting these recommendations are based on non-randomised comparative studies or case series, because randomised controlled clinical trials or meta-analyses are not available for this disease. Results and conclusions. Desmopressin (DDAVP) is the treatment of choice for patients with type 1 VWD with FVIII and VWF levels of 10 U/dL or more, while VWF/FVIII concentrates are indicated for those who are unresponsive or insufficiently responsive to DDAVP (severe type 1, type 2 and 3 VWD). VWF concentrates devoid of FVIII, not yet licensed in Italy, may be considered for short-term prophylaxis in elective surgery or for long-term secondary prophylaxis.
| Original language | English |
|---|---|
| Pages (from-to) | 117-126 |
| Number of pages | 10 |
| Journal | Blood Transfusion |
| Volume | 7 |
| Issue number | 2 |
| DOIs | |
| Publication status | Published - 2009 |
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Keywords
- Desmopressin
- Von Willebrand disease
- Von Willebrand factor
ASJC Scopus subject areas
- Hematology
- Immunology and Allergy
Cite this
Evidence-based recommendations on the treatment of von Willebrand disease in Italy. / Mannucci, Pier O.; Franchini, Massimo; Castaman, Giancarlo; Federici, Augusto B.
In: Blood Transfusion, Vol. 7, No. 2, 2009, p. 117-126.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Evidence-based recommendations on the treatment of von Willebrand disease in Italy
AU - Mannucci, Pier O.
AU - Franchini, Massimo
AU - Castaman, Giancarlo
AU - Federici, Augusto B.
PY - 2009
Y1 - 2009
N2 - Background. von Willebrand disease (VWD) is the most common hereditary bleeding disorder affecting both males and females. It arises from quantitative or qualitative defects of von Willebrand factor (VWF) and causes bleeding of mucous membranes and soft tissues. The aim of treatment is to correct the dual defect of haemostasis caused by the abnormal/reduced VWF and the concomitant deficiency of factor VIII (FVIII). Material and methods. This document contains evidence-based recommendations for the management of VWD compiled by AICE (the Italian Association of Haemophilia Centres). All the evidence supporting these recommendations are based on non-randomised comparative studies or case series, because randomised controlled clinical trials or meta-analyses are not available for this disease. Results and conclusions. Desmopressin (DDAVP) is the treatment of choice for patients with type 1 VWD with FVIII and VWF levels of 10 U/dL or more, while VWF/FVIII concentrates are indicated for those who are unresponsive or insufficiently responsive to DDAVP (severe type 1, type 2 and 3 VWD). VWF concentrates devoid of FVIII, not yet licensed in Italy, may be considered for short-term prophylaxis in elective surgery or for long-term secondary prophylaxis.
AB - Background. von Willebrand disease (VWD) is the most common hereditary bleeding disorder affecting both males and females. It arises from quantitative or qualitative defects of von Willebrand factor (VWF) and causes bleeding of mucous membranes and soft tissues. The aim of treatment is to correct the dual defect of haemostasis caused by the abnormal/reduced VWF and the concomitant deficiency of factor VIII (FVIII). Material and methods. This document contains evidence-based recommendations for the management of VWD compiled by AICE (the Italian Association of Haemophilia Centres). All the evidence supporting these recommendations are based on non-randomised comparative studies or case series, because randomised controlled clinical trials or meta-analyses are not available for this disease. Results and conclusions. Desmopressin (DDAVP) is the treatment of choice for patients with type 1 VWD with FVIII and VWF levels of 10 U/dL or more, while VWF/FVIII concentrates are indicated for those who are unresponsive or insufficiently responsive to DDAVP (severe type 1, type 2 and 3 VWD). VWF concentrates devoid of FVIII, not yet licensed in Italy, may be considered for short-term prophylaxis in elective surgery or for long-term secondary prophylaxis.
KW - Desmopressin
KW - Von Willebrand disease
KW - Von Willebrand factor
UR - http://www.scopus.com/inward/record.url?scp=66349136610&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=66349136610&partnerID=8YFLogxK
U2 - 10.2450/2008.0052-08
DO - 10.2450/2008.0052-08
M3 - Article
C2 - 19503633
AN - SCOPUS:66349136610
VL - 7
SP - 117
EP - 126
JO - Blood Transfusion
JF - Blood Transfusion
SN - 1723-2007
IS - 2
ER -