Latin-American Von Willebrand Disease Registry

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

Study Overview

• Status: Unknown
• Conditions: Von Willebrand Disease
• Intervention / Treatment: Other: Observation

Detailed Description

von Willebrand disease (VWD) is the most common autosomal bleeding disorder, mostly inherited as dominant trait. VWD is due to deficiency/abnormality of von Willebrand factor (VWF). The prevalence of VWD is unknown, but estimated as 0.1% to 1% of the general population. Although the autosomal inheritance pattern would suggest an equal distribution of male and female patients, the disease is diagnosed in more females because of female-specific hemostatic challenges: menses, ovulation, pregnancy and childbirth. Diagnosis of VWD is made by assessing personal and family history of bleeding, physical examination and completed with specific laboratory tests.

There is limited information on the epidemiology of VWD in developing countries. Some countries in Latin America have registries of severe disease that, although it is the rarest form, carries the highest costs for regional health systems. So that the prevalence of clinical symptoms and laboratory features of the disease as well as the management of the disease in Latin America is unknown.

The present project aims to establish a network of centers and professionals with the objective to register and investigate all patients with VWD in Latin America, using a database available online common to all, to gain understanding about phenotype, genotype and management of VWD in the region.

• Study Type: Observational
• Enrollment (Anticipated): 500

Contacts and Locations

• Study Contact: Name: Analia Sanchez Luceros, PhD, MD; Phone Number: +5491152203235; Email: sanchezluceros@gmail.com
• Study Contact Backup: Name: Analia Kinen; Email: analiakinen@hotmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: N/A
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients with von Willebrand disease

Description

Inclusion Criteria:

• 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

Exclusion Criteria:

• Patient without consent to participate

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Cross-Sectional

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Subjects with von Willbrand Disease Acquired	Other: Observation; No interventions planned: treatment of patients at the discretion of the treating/responsible physician
Subjects with von Willbrand Disease Congenital	Other: Observation; No interventions planned: treatment of patients at the discretion of the treating/responsible physician

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo	For many women with VWD, pregnancy is a time of few bleeding problems. Women with Type 3 von Willebrand disease seem to have more frequent miscarriages, especially during the first trimester. 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).	Through study completion, an average of 2 years

Collaborators and Investigators

• Sponsor: Academia Nacional de Medicina

Study record dates

Study Major Dates

• Study Start (Anticipated): February 1, 2020
• Primary Completion (Anticipated): December 1, 2022
• Study Completion (Anticipated): December 1, 2022

Study Registration Dates

• First Submitted: February 7, 2020
• First Submitted That Met QC Criteria: February 19, 2020
• First Posted (Actual): February 21, 2020

Study Record Updates

• Last Update Posted (Actual): February 21, 2020
• Last Update Submitted That Met QC Criteria: February 19, 2020
• Last Verified: February 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases
• Other Study ID Numbers: Protocol_3081

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Sharing Supporting Information Type: Study Protocol; Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No