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
Studieoversikt
Detaljert beskrivelse
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.
Studietype
Registrering (Forventet)
Kontakter og plasseringer
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
- Eldre voksen
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Prøvetakingsmetode
Studiepopulasjon
Beskrivelse
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
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Observasjonsmodeller: Kohort
- Tidsperspektiver: Tverrsnitt
Kohorter og intervensjoner
Gruppe / Kohort |
Intervensjon / Behandling |
|---|---|
|
Subjects with von Willbrand Disease Acquired
|
Ingen intervensjoner planlagt: behandling av pasienter etter skjønn fra behandlende/ansvarlig lege
|
|
Subjects with von Willbrand Disease Congenital
|
Ingen intervensjoner planlagt: behandling av pasienter etter skjønn fra behandlende/ansvarlig lege
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Register of VWD patients in Latin America
Tidsramme: assessed up to 33 months
|
Clinical presentation in hereditary/acquired VWD.
Phenotype and genetic diagnosis.
|
assessed up to 33 months
|
|
Registration of the bleeding history
Tidsramme: 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.
|
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
Tidsramme: 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).
|
Until the end of the registry, an average of 33 months.
|
|
Adverse Events: Number of patients with bleeding events
Tidsramme: 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.
|
until the end of the registry, an average of 33 months.
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo
Tidsramme: 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).
|
Through study completion, an average of 2 years
|
Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart (Forventet)
Primær fullføring (Forventet)
Studiet fullført (Forventet)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- Protocol_3081
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-deling Støtteinformasjonstype
- Studieprotokoll
- Klinisk studierapport (CSR)
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Studerer et amerikansk FDA-regulert enhetsprodukt
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