Our primary aim is to investigate the current bleeding tendency of patients with VWD.
ID
Source
Brief title
Condition
- Coagulopathies and bleeding diatheses (excl thrombocytopenic)
- Blood and lymphatic system disorders congenital
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The major-bleeding rate: bleeding that require blood transfusion, desmopressine
or factor concentrates or bleeding that lead to death.
Secondary outcome
Secundary outcomes are the minor-bleeding rate (all bleeding that do not fulfil
the criteria for major-bleeding), bleeding during surgery, Von Willebrand
factor levels and the ISTH-BAT score.
Background summary
Von Willebrand disease (VWD) is the most common inherited bleeding disorder,
and is characterized by a defective platelet adhesion and aggregation. VWD is
caused by a reduced (type 1), an abnormal function (type 2) or a complete
absence (type 3) of von Willebrand factor (VWF).
The diagnosis of VWD is based on the evaluation of the personal
bleeding history and family history, followed by detailed laboratory
evaluation. To assess the bleeding phenotype, the International Society for
Thrombosis and Hemostasis Bleeding Assessment Tool (ISTH-BAT) is recommended as
bleeding score. The laboratory evaluation of VWD consists out of measurement of
VWF antigen (VWF:Ag), and VWF activity: VWF-mediated platelet agglutination
(VWF:RCo) and the binding of VWF to collagen (VWF:CB), and the coagulant
activity of Factor VIII (FVIII:C).
In recent years large retrospective cohort studies have provided
valuable insights on the clinical presentation, bleeding phenotype, quality of
life, diagnostics, genetics and treatment of patients with VWD. All large
retrospective cohort studies have assessed the bleeding phenotype of patients
with VWD using bleeding scores or retrospective questionnaires. Bleeding scores
calculate the sum of all bleeding episodes during lifetime. Therefore, they do
not provide information on the change of bleeding tendency. If a patient had a
period in his or her lifetime in which he or she had many bleeding episodes,
then the bleeding score is high. Though, the patient could have had those
bleeds 30 years ago and did not have a bleeding episode since then. Therefore,
bleeding scores do not provide information on the current bleeding phenotype of
VWD patients, and prospective studies are needed to investigate the current
bleeding tendency of patients with VWD.
Study objective
Our primary aim is to investigate the current bleeding tendency of patients
with VWD.
Study design
We will conduct a nationwide, multi-center, observational, prospective cohort
study in all VWD patients in the Netherlands.
This study has 2 important elements; patients will visit their own hemophilia
treatment center for study inclusion and patients will be followed-up for a
period of 2 years, during which they will fill in online questionnaires each 3
months.
Study burden and risks
This study has an observational character. The only interventions in this study
are the questionnaires, a blood drawal at study inclusion, a blood pressure
measurement and a capillary nail fold measurement. We consider the risks of
this study to be negligible and the burden to be minimal. The total burden of
this study is approximately 2 to 3 hours during a period of 2 years.
Wytemaweg 80
Rotterdam 3015 CN
NL
Wytemaweg 80
Rotterdam 3015 CN
NL
Listed location countries
Age
Inclusion criteria
- Historically lowest VWF:Ag and/or VWF:Act and/or VWF:CB <= 0.30 IU/mL and/or
FVIII:C <= 0.40 IU/mL.
- Treatment at a Hemophilia treatment center in the Netherlands
- All types of VWD
- All ages
Exclusion criteria
- Other bleeding disorders present
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | Identificatienummer volgt |
| CCMO | NL62238.078.18 |