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

NCT ID: NCT00510042

Last Updated: 2010-04-26

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 1100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2007-07-31
• Study Completion Date: 2009-09-30

Brief 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.

Detailed 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.

Conditions

Von Willebrand Disease

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: CROSS_SECTIONAL

Eligibility Criteria

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Foundation Haemophilia

UNKNOWN

Sponsor Role: collaborator

CSL Behring

INDUSTRY

Sponsor Role: collaborator

Erasmus Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Erasmus University Medical Center

Principal Investigators

Eva M de Wee, MD

Role: STUDY_DIRECTOR

Erasmus Medical Center

Frank WG Leebeek, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Erasmus Medical Center

Karin Fijn van Draat, MD, PhD

Role: STUDY_CHAIR

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

Jeroen CJ Eikenboom, MD, PhD

Role: STUDY_CHAIR

Leiden University Medical Center

Arja de Goede-Bolder, MD

Role: STUDY_CHAIR

Erasmus Medical Center

Eveline P Mauser-Bunschoten, MD, PhD

Role: STUDY_CHAIR

Van Creveldkliniek, University Medical Center Utrecht

Karina Meijer, MD, PhD

Role: STUDY_CHAIR

University Medical Center Goningen

Britta Laros-van Gorkom, MD, PhD

Role: STUDY_CHAIR

University Medical Center St. Radboud Nijmegen

Johanna G van der Bom, PhD

Role: STUDY_CHAIR

Leiden University Medical Center

Manon A Degenaar-Dujardin, BA

Role: STUDY_CHAIR

Organization for Hemophilia Patients

Locations

Academic Medical Center Amsterdam

Amsterdam, , Netherlands

VU University Medical Center

Amsterdam, , Netherlands

Amphia Hospital

Breda, , 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

Haga Hospital

The Hague, , Netherlands

University Medical Center Utrecht Van Creveldkliniek

Utrecht, , Netherlands

Countries

Netherlands

References

Mannucci PM. Treatment of von Willebrand's Disease. N Engl J Med. 2004 Aug 12;351(7):683-94. doi: 10.1056/NEJMra040403. No abstract available.

Reference Type: BACKGROUND

PMID: 15306670 (View on PubMed)

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. No abstract available.

Reference Type: BACKGROUND

PMID: 10959685 (View on PubMed)

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.

Reference Type: BACKGROUND

PMID: 8052974 (View on PubMed)

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. doi: 10.1111/j.1538-7836.2006.01847.x.

Reference Type: BACKGROUND

PMID: 16634745 (View on PubMed)

Federici AB. Clinical diagnosis of von Willebrand disease. Haemophilia. 2004 Oct;10 Suppl 4:169-76. doi: 10.1111/j.1365-2516.2004.00991.x.

Reference Type: BACKGROUND

PMID: 15479393 (View on PubMed)

Other Identifiers

MEC-2007-063

Identifier Type: -

Identifier Source: org_study_id