Performance Evaluation of Von Willebrand:Collagen-Binding Assays to Diagnose Von Willebrand Factor Deficiency in Patients With Increased Risk of Bleeding

NCT02792205 | Completed

• 1 other identifier: RC16_0028
• Study Type: observational
• Target: 70
• Locations: 1 country
• Sites: 2

Brief Summary

Von Willebrand Disease (VWD) is defined as an inherited bleeding disorder that is caused by deficiency or dysfunction of von Willebrand factor (VWF), a plasma protein that mediates the initial adhesion of platelets at sites of vascular injury and also binds and stabilizes blood clotting factor VIII (FVIII) in the circulation. The most severe forms of VWD are usually easy to diagnose (obvious hemorrhagic symptoms and major VWF deficiency), whereas the mild forms of the disease are still difficult to confirm. It is indeed reported that about 1% of the population carry mild biological VWF deficiency without any bleeding tendency and any "actual disease". On the contrary, some patients with severe bleeding history can carry a true VWF abnormality, well-confirmed by genetic studies, without any VWF deficiency when evaluated with standard biological methods, such as Ristocetin Cofactor activity (VWF:RCo). However, in these patients, the use of alternative methods, such as PFA-100 (Platelet Fonction Analyzer-100), the study of Factor VIII (FVIII:C) to VWF (FVIII:C/VWF) ratio or the evaluation of VWF activity using more specialized methods such as VWF:CB (VWF-Collagen Binding) assay can detect the VWF deficiency and possible hemorrhagic predisposition. In this project, the investigators plan to assess the performance of VWF:CB in the diagnosis of VWF deficiency in patients with unexplained bleeding history.

Conditions

Von Willebrand Disease

Keywords

Willebrand factor deficiency; hematology; Willebrand:Collagen-binding

Outcome Measures

Primary Outcomes (1)

• Von Willebrand factor levels measured with Von Willebrand factor: Collagen-Binding methods

  Deficiency is defined when VWF level is \< 50IU/dL, as usually defined by Favaloro E.J. (2000). A composite reference standard (CRS) will be used to improve the imperfect Gold Standard (VWF: RCo). CRS will be defined as being positive if either VWF: RCo, VWF:Ag, PFA-100 or FVIII:C will be positive and negative otherwise.

  Up to 1 year

Interventions

Non interventional study OTHER

no intervention

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study plans to enroll 200 patients (100 per Center) in a 2-year period. The recruitment will occur in standard consultation activity of the 2 Hemostasis Centers of Dijon and Nantes. As each Center carries out about 1200 consultations/year, this projection appears as feasible, since a majority of them are induced by hemorrhagic profile. Such a number of patients will allow reliable statistical analysis.

You may qualify if:

• Patient with bleeding history sent to the Hemophilia Treatment Center in Dijon or Nantes University Hospital with abnormal bleeding score (\>3 in men and \>5 in women
• Patient who has provided a signed consent to participate at this study and for blood sampling
• Affiliation with French social security system
• Minors will not be included in the study.
• On-going pregnancy and postpartum period (3 months after delivery)
• Substitutive treatment with coagulation factor concentrates or desmopressin administration within 10 days before sampling.
• Ongoing infectious or inflammatory disease that can modify VWF levels.
• Diagnosis of obvious hemostasis disorder

Sponsors & Collaborators

• Nantes University Hospital: lead

Study Sites (2)

Dijon University Hospital

Dijon, 21079, France

Location

Nantes University Hospital

Nantes, 44093, France

Location

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Marc Trossaërt, Dr

  Nantes University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2016
• First Posted: June 7, 2016
• Study Start: February 22, 2017
• Primary Completion: June 30, 2021
• Study Completion: June 30, 2021
• Last Updated: June 10, 2022

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)