ABSTRACT
The aim of treatment of von Willebrand disease (VWD) is to correct the dual defect
of hemostasis (i.e., the abnormal platelet adhesion due to reduced and/or dysfunctional
von Willebrand factor [VWF] and the abnormal coagulation expressed by low levels of
factor [F] VIII). Desmopressin acetate (DDAVP) is the treatment of choice for type
1 VWD because it can induce release of normal VWF from cellular compartments. Prospective
studies on biological response versus clinical efficacy of DDAVP in VWD types 1 and
2 are in progress to explore its benefits and limits as a therapeutic option. In type
3 and in severe forms of type 1 and 2 VWD, DDAVP is not effective, and for these patients
plasma virally inactivated concentrates containing VWF and FVIII are the mainstay
of treatment. Several intermediate- and high-purity VWF/FVIII concentrates are available
and have been shown to be effective in clinical practice (bleeding and surgery). New
VWF products almost devoid of FVIII are now under evaluation in clinical practice.
Although thrombotic events are rare in VWD patients receiving repeated infusions of
concentrates, there is some concern that sustained high FVIII levels may increase
risk of postoperative venous thromboembolism. Dosage and timing of VWF/FVIII administrations
should be planned to keep the FVIII level between 50 and 150 IU/dL. Appropriate dosage
and timing in repeated infusions are also very important in patients exposed to secondary
long-term prophylaxis for recurrent bleedings.
KEYWORDS
von Willebrand disease - von Willebrand factor - desmopressin - factor VWF/VIII concentrates
- efficacy and safety of concentrates - secondary long-term prophylaxis
REFERENCES
augusto.federici@unimi.it
