National Study of Moderate and Severe Von Willebrand Disease in the Netherlands

Von Willebrand Disease Clinical Trial

— WiN  

Official title:

National Study of Moderate and Severe Von Willebrand Disease in the Netherlands

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00510042
• Other study ID #: MEC-2007-063
• Status: Completed
• Phase: N/A
• First received: July 31, 2007
• Last updated: April 23, 2010
• Start date: July 2007
• Est. completion date: September 2009

Study information

• Verified date: April 2010
• Source: Erasmus Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: Medical Ethics Review Committee (METC)
• Study type: Observational

Clinical Trial Summary

The objective of this study is to assess the clinical presentation, the treatment and the complications of the disease and treatment in moderate and severe von Willebrand disease. Another goal is to investigate the influence of von Willebrand disease on quality of life.

Description:

The incidence of von Willebrand disease in the Netherlands is unknown, but the occurrence of all types of von Willebrand disease together is estimated at 1:100-1:200 individuals. Most patients have mild von Willebrand disease, mostly type 1. For the more severe forms of von Willebrand disease the incidence in unclear, but the expectation is, that there are at least 500-1500 patients. In the Netherlands, an unique situation exists for the treatment of patients with hemophilia and related coagulation disorders. In 2000 a hemophilia management policy was set up by the Ministry of Health, which stated that the care for these patients should be concentrated in 13 Hemophilia Treatment Centers (HTC). The representatives of the attending centers, the hemophilia specialists, are organized in the NVHB, the Dutch society of Hemophilia treaters. In the hemophilia management policy is stated that all patients with a coagulation disorder dependent of replacement products must be treated in a HTC or under responsibility of a HTC. These patients are seen in a HTC at least once a year.

Because all moderate and severe von Willebrand patients in the Netherlands are known in HTC, it is possible to register and study this population. This is imperative to the research of von Willebrand disease. Because the moderate and severe forms of von Willebrand disease are rare, it is impossible for an individual center to perform research of moderate severe and severe von Willebrand disease. Therefore a national approach is necessary.

For optimal care of patients with the moderate and severe form of von Willebrand disease a better understanding of symptoms, diagnostics, treatment and complications of treatment is necessary. The present study aims to register and investigate all patients in the Netherlands with moderate and severe von Willebrand disease to gain understanding of the clinical presentation, the treatment and the complications of treatment in moderate and severe von Willebrand disease. Another goal is to investigate the influence of von Willebrand disease on quality of life.

To answer these questions a questionnaire is developed, which will be sent to the study population. In addition, blood will be drawn for von Willebrand factor measurement and plasma and DNA will be stored. The relationship between laboratory parameters, (including von Willebrand factor, FVIII and prothrombotic coagulation disorders) and the clinical phenotype in patients with moderate and severe von Willebrand disease will be studied. We will assess the effect of the laboratory parameters on both the severity of bleeding tendency, and the possible protecting effect on the risk of arterial thrombosis. In the future mutation analysis of the VWF gene will be performed in patients with moderate and severe von Willebrand disease in the Netherlands.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1100
• Est. completion date: September 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. haemorrhagic symptoms or a family history of von Willebrand disease

2. vWF antigen = 30%, lowest measurement counts and/or vWF activity (vWF:RCo of vWF:CB) = 30%, lowest measurement counts and/or FVIII:C = 40 %, lowest measurement counts

3. being known in a hemophilia treatment center or if only the diagnose is made in a hemophilia treatment center this must be done after 1987

Exclusion Criteria:

1. Hemophilia A

2. Carriership of hemophilia A

3. No informed consent

Study Design

Observational Model: Cohort, Time Perspective: Cross-Sectional

Related Conditions & MeSH terms

• Von Willebrand Disease
• Von Willebrand Diseases

Locations

Country	Name	City	State
Netherlands	Academic Medical Center Amsterdam	Amsterdam
Netherlands	VU University Medical Center	Amsterdam
Netherlands	Amphia Hospital	Breda
Netherlands	Haga Hospital	Den Haag
Netherlands	Maxima Medical Center	Eindhoven
Netherlands	University Medical Center Groningen	Groningen
Netherlands	Kennemer Hospital	Haarlem
Netherlands	Leiden University Medical Center	Leiden
Netherlands	Academic Hospital Maastricht	Maastricht
Netherlands	University Medical Center St. Radboud	Nijmegen
Netherlands	Erasmus University Medical Center	Rotterdam
Netherlands	University Medical Center Utrecht Van Creveldkliniek	Utrecht

Sponsors (3)

Lead Sponsor	Collaborator
Erasmus Medical Center	CSL Behring, Foundation Haemophilia

Country where clinical trial is conducted

Netherlands, 

References & Publications (5)

Federici AB. Clinical diagnosis of von Willebrand disease. Haemophilia. 2004 Oct;10 Suppl 4:169-76. Review. — View Citation

Mannucci PM. Treatment of von Willebrand's Disease. N Engl J Med. 2004 Aug 12;351(7):683-94. Review. — View Citation

Sadler JE, Mannucci PM, Berntorp E, Bochkov N, Boulyjenkov V, Ginsburg D, Meyer D, Peake I, Rodeghiero F, Srivastava A. Impact, diagnosis and treatment of von Willebrand disease. Thromb Haemost. 2000 Aug;84(2):160-74. Review. — View Citation

Sadler JE. A revised classification of von Willebrand disease. For the Subcommittee on von Willebrand Factor of the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 1994 Apr;71(4):520-5. — View Citation

Tosetto A, Rodeghiero F, Castaman G, Goodeve A, Federici AB, Batlle J, Meyer D, Fressinaud E, Mazurier C, Goudemand J, Eikenboom J, Schneppenheim R, Budde U, Ingerslev J, Vorlova Z, Habart D, Holmberg L, Lethagen S, Pasi J, Hill F, Peake I. A quantitative analysis of bleeding symptoms in type 1 von Willebrand disease: results from a multicenter European study (MCMDM-1 VWD). J Thromb Haemost. 2006 Apr;4(4):766-73. — View Citation