Latin-American Von Willebrand Disease Registry

• Conditions: Von Willebrand Disease
• Interventions: Other: Observation

• Registration Number: NCT04279717
• Lead Sponsor: Academia Nacional de Medicina

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

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 Timeline

• Status Verified: 2020-02
• Study Start: 2020-02
• Study First Submitted: 2020-02-07
• Study First Submitted QC: 2020-02-19
• Last Update Submitted: 2020-02-19
• Study First Posted: 2020-02-21
• Last Update Posted: 2020-02-21
• Primary Completion: 2022-12
• Study Completion: 2022-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 500

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Subjects with von Willbrand Disease Acquired	Observation	-
Subjects with von Willbrand Disease Congenital	Observation	-

Primary Outcome Measures

Name	Time	Method
Register of VWD patients in Latin America	assessed up to 33 months	Clinical presentation in hereditary/acquired VWD. Phenotype and genetic diagnosis.
Adverse Events: Number of patients with bleeding events	until the end of the registry, an average of 33 months.	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.
Registration of the bleeding history	From date of selection until the date registration, assessed up to 33 months.	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.
Response to Treatment: Follow up of FVIII, VWF:Ag and VWF:RCo	Until the end of the registry, an average of 33 months.	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).

Secondary Outcome Measures

Name	Time	Method
Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo	Through study completion, an average of 2 years	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).