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
연구 개요
상세 설명
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.
연구 유형
등록 (예상)
연락처 및 위치
참여기준
자격 기준
공부할 수 있는 나이
- 어린이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
설명
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
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: 보병대
- 시간 관점: 단면
코호트 및 개입
그룹/코호트 |
개입 / 치료 |
|---|---|
|
Subjects with von Willbrand Disease Acquired
|
계획된 개입 없음: 치료/책임 의사의 재량에 따라 환자 치료
|
|
Subjects with von Willbrand Disease Congenital
|
계획된 개입 없음: 치료/책임 의사의 재량에 따라 환자 치료
|
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Register of VWD patients in Latin America
기간: 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
기간: 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
기간: 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
기간: 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.
|
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo
기간: 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
|
공동 작업자 및 조사자
연구 기록 날짜
연구 주요 날짜
연구 시작 (예상)
기본 완료 (예상)
연구 완료 (예상)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
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