REAL-LIFE DATA OF CONSTITUTIONAL VON WILLEBRAND DISEASE IN WESTERN FRANCE (HOPSCOTcH-WILL)

NCT03875924 | Completed

• 1 other identifier: RC19_0075
• Study Type: observational
• Target: 926
• Locations: 1 country
• Sites: 5

Brief Summary

Von Willebrand disease (VWD) is the most common constitutional bleeding disorder in the world, caused by missing or defective von Willebrand factor (VWF). In France, VWD affects approximatively 7,000 patients. There are many types of VWD. The severest forms are characterized by the occurrence of extremely serious bleedings, requiring in-stays with clotting factors (CF) treatments in specialized hospital units and/or an ambulatory substitutive therapy; both of them are highly expensive. In France, Hemostasis Treatment Centers (HTC) have the opportunity to record these kinds of data in a database called NHEMO (Net-Hemostasis = care database for constitutional bleeding disorders). Further ahead, the data can be coded, dumped into and extracted from the research database BERHLINGO and analyzed. The HOPSCOTcH-WILL study will be a retrospective, non-interventional, multicenter (national) cohort study \& will provide an overview of the real-life management of patients with VWD in western France requiring a substitutive treatment with VWF, as well as a description of the characteristics of their hemorrhagic events. Model : Observationnal, real world evidence study. Time Horizon : 2015-2018. HTC (France): Western University Hospitals (BERHLINGO network) = Nantes University Hospital (promotion), Angers University Hospital, Brest University Hospital, Le Mans Regional Hospital \& Rennes University Hospital

Conditions

Von Willebrand Diseases

Outcome Measures

Primary Outcomes (1)

• Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients of any severity (in and outpatients care consumptions)

  Every treatment over 48 months

Secondary Outcomes (8)

• Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by type of VWD (in and outpatients care consumptions)

  Every treatment over 48 months

• Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, according the history of inhibitor (in and outpatients care consumptions)

  Every treatment over 48 months

• Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by regimen of treatment (in and outpatients care consumptions)

  Every treatment over 48 months

• Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by type of treatment (pdVWF, FVIII, pdVWF/FVIII, desmopressin)

  Every treatment over 48 months

• Consumptions of clotting factors/desmopressine for the treatment of severe hemorrhagic events in VWD patients, by type of hemorrhage

  Every treatment over 48 months

Study Arms (1)

VWD BERHLINGO

Patients with constitutional von Willebrand Disease, of any severity, with or without inhibitor followed in one of the investigator centers

Other: VWD treatments

Interventions

VWD treatments OTHER

Modalities and types of treatments in VWD patients for severe hemorrhagic events: INN, quantities, duration of treatment

VWD BERHLINGO

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

On 16th July 2018, 919 constitutional VWD\* patients are included in the NHEMO database for the 5 Western France BERHLINGO HTC = Angers, Brest, Le Mans, Nantes \& Rennes. In case of hemorrhagic events: * 16.4% (n=151) are likely to be treated with desmopressin only, * 20.8% (n=191) with CF only - i.e. von Willebrand Factor (VWF) and/or Factor VIII (FVIII) and /or by-passing agents -, * 21.6% (n=199) with desmopressin and/or CF. To be taken into account: 41.2% of patients have no treatment mentioned or are in a phase of evaluation of their modalities of treatment (=desmopressin test to be done or desmopressin treatment to be debated before use, essentially). It is estimated that out of these potential treated patients, 300 could have been treated with CF at least once during the 4-years period. \*According to the CRMW criteria for constitutional VWD

You may qualify if:

• Constitutional VWD patient, of any severity, with or without inhibitors (according to the CRMW criteria\*),
• Patient included in the research database BERHLINGO
• Patient treated or not by desmopressin or VWF/FVIII/by-passing agents available on the French market (at baseline)
• Patient who agrees to participate in the HOPSCOTcH and followed in one of the 5 investigator HTC
• Patient who are not under guardianship
• CRMW criteria for constitutional VWD definition:
• Severe forms: VWF:Ag and VWF:RCo \<5 UI/dL VWD 2A or 2M: VWF:RCo/VWF:Ag \< 0.7 and/or ratio VWF:CB/VWF:Ag \< 0.7 VWD 2B: unexplained thrombopenia and/or positive RIPA \< 0.8 mg/mL (for any value of VWF:RCo/VWF:Ag) VWD 2N: FVIII:C/VWF:Ag \< 0.6 and reduced to very reduced VWF:FVIIIB VWF:Ag \<30 UI/dL (in the absence of every previous criteria)

You may not qualify if:

• Patients Under guardianship

Sponsors & Collaborators

• Nantes University Hospital: lead
• CHU de Brest: collaborator
• University Hospital, Angers: collaborator
• CH Le Mans: collaborator
• Rennes University Hospital: collaborator

Study Sites (5)

Angers University Hospital

Angers, France

Location

Brest University Hospital

Brest, France

Location

Le Mans Regional Hospital

Le Mans, France

Location

Nantes University Hospital

Nantes, France

Location

Rennes University Hospital

Rennes, France

Location

Related Publications (1)

• Horvais V, Beurrier P, Cussac V, Pan-Petesch B, Schirr-Bonnans S, Rose J, Bayart S, Ternisien C, Fouassier M, Sigaud M, Babuty A, Drillaud N, Guillet B, Trossaert M; BERHLINGO Consortium. Key Drivers of Coagulation Factor Use in Von Willebrand Disease During Hospitalization: An Overview of the French BERHLINGO Cohort. Clin Drug Investig. 2024 Jan;44(1):35-49. doi: 10.1007/s40261-023-01323-1. Epub 2023 Nov 30.

  PMID: 38036930 DERIVED

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 Design

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

Study Record Dates

• First Submitted: March 13, 2019
• First Posted: March 15, 2019
• Study Start: November 13, 2019
• Primary Completion: December 31, 2019
• Study Completion: December 31, 2019
• Last Updated: May 19, 2020

Record last verified: 2020-05

Locations

FR (5)