Clinical trial NCT04279717: Latin-American Von Willebrand Disease Registry
Official title: Latin-American Von Willebrand Disease Registry
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Von Willebrand Disease
Lead sponsor: Academia Nacional de Medicina
Sponsor class: OTHER
First posted: 2020-02-21
Enrollment: 500
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with von Willebrand disease

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.

Eligibility criteria: 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

Primary outcomes:
measure: Register of VWD patients in Latin America
timeFrame: assessed up to 33 months
measure: Registration of the bleeding history
timeFrame: From date of selection until the date registration, assessed up to 33 months.
measure: Response to Treatment: Follow up of FVIII, VWF:Ag and VWF:RCo
timeFrame: Until the end of the registry, an average of 33 months.
measure: Adverse Events: Number of patients with bleeding events
timeFrame: until the end of the registry, an average of 33 months.
Secondary outcomes:
measure: Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo
timeFrame: Through study completion, an average of 2 years
Other outcomes:
Locations:
Study officials:
References: