Latin-American Von Willebrand Disease Registry

NCT04279717 | Unknown

• 1 other identifier: Protocol_3081
• Study Type: observational
• Target: 500
• Locations: 0 countries
• Sites: N/A

Brief Summary

Establish a Latin-American network of centers and professionals with the aim of:

• To register VWD patients in retrospective/prospective study, using a database, available online, common to all
• To register the bleeding history, the treatment and the events of VWD patients in the region
• To investigate the influence of VWD on quality of life

Conditions

Von Willebrand Disease

Outcome Measures

Primary Outcomes (4)

• Register of VWD patients in Latin America

  Clinical presentation in hereditary/acquired VWD. Phenotype and genetic diagnosis.

  assessed up to 33 months

• Registration of the bleeding history

  Bleeding history is an essential component in the diagnosis of von Willebrand disease (VWD). ISTH Bleeding Assessment Tool (ISTH-BAT) is used to assist the diagnosis.

  From date of selection until the date registration, assessed up to 33 months.

• Response to Treatment: Follow up of FVIII, VWF:Ag and VWF:RCo

  The aim of therapy is to correct the dual hemostatic defect, due to defective platelet adhesion-aggregation and abnormal coagulation due to Factor VIII (FVIII) deficiency. The choice of treatment depends on a number of factors, including the severity of the bleed, the procedure planned, the subtype and severity of the disease and the age and morbidity of the patient. The evaluation of the response to the treatment is going to be through the measure of FVIII, vWF Antigen (VWF:Ag) and vWF ristocetin cofactor (vWF:RCo).

  Until the end of the registry, an average of 33 months.

• Adverse Events: Number of patients with bleeding events

  Bleeding disorders and their treatment impact on patients, especially in women, can affect the everyday life of patients and their families. Measure of number of bleeding events, laboratory results such as Sodium.

  until the end of the registry, an average of 33 months.

Secondary Outcomes (1)

• Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo

  Through study completion, an average of 2 years

Study Arms (2)

Subjects with von Willbrand Disease Acquired

Other: Observation

Subjects with von Willbrand Disease Congenital

Other: Observation

Interventions

Observation OTHER

No interventions planned: treatment of patients at the discretion of the treating/responsible physician

Subjects with von Willbrand Disease Congenital

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with von Willebrand disease

You may qualify if:

• Historically lowest VWF:Ag and/or VWF:RCo and/or VWF:CB \< 0.50 IU/ml and/or FVIII:C \< 0.50 IU/ml
• All types of VWD
• All ages

You may not qualify if:

• Patient without consent to participate

Sponsors & Collaborators

• Academia Nacional de Medicina: lead

Biospecimen

Retention: SAMPLES WITH DNA

Plasma, DNA

MeSH Terms

Conditions

von Willebrand Diseases

Interventions

Observation

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

Intervention Hierarchy (Ancestors)

Methods; Investigative Techniques

Central Study Contacts

Analia Sanchez Luceros, PhD, MD

CONTACT

+5491152203235; sanchezluceros@gmail.com

Analia Kinen

CONTACT

analiakinen@hotmail.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 7, 2020
• First Posted: February 21, 2020
• Study Start: February 1, 2020
• Primary Completion: December 1, 2022
• Study Completion: December 1, 2022
• Last Updated: February 21, 2020

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR