Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador

Hemophilia A Clinical Trial

— VWD  

Official title:

Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01589848
• Other study ID #: 001
• Secondary ID: UAzuay
• Status: Completed
• Phase: N/A
• First received: April 30, 2012
• Last updated: July 2, 2013
• Start date: March 2013
• Est. completion date: June 2013

Study information

• Verified date: July 2013
• Source: Universidad del Azuay
• Contact: n/a
• Is FDA regulated: No
• Health authority: Ecuador: Comité de Bioética, Universidad San Francisco de Quito
• Study type: Observational

Clinical Trial Summary

1. Hypothesis

a. There are patients with von Willebrand Disease in Cuenca.

2. Primary question

a. How many women referred with a history of bleeding may have von Willebrand disease?

3. Secondary

1. Associations between the bleeding score and initial laboratory studies

2. What are the differences on subgroups of enrolled patients with the bleeding score?

4. Ancillary

1. What is the clinical and socio-economic status of women with von Willebrand Disease in Cuenca?

2. What is the clinical and socio-economic status of patients with Hemophilia in Cuenca?

Description:

The incidence of hemophilia A is 1 in 10,000 and the one of hemophilia B is 1 in 30,000 live born males. As the most frequent coagulopathy in the world, von Willebrand disease (vWD) can affect 1 in 1000 children of both genders. The prevalence of vWD is 1-2% and it may be as high as 5-15% among women with menorrhagia. So vWD can be present in 1 of every 100 to 500 individuals.

A world survey published that Ecuador, with a population of 14,790,608 people, has 238 patients with hemophilia, 45 people with von Willebrand disease (vWD) and 13 patients with other bleeding disorders including rare factor deficiencies and platelet disorders.

The Ecuadorian census of 2010 indicates that Cuenca district (canton) has 505,585 inhabitants. According to a local reference, Cuenca has no reports of vWD and it has registered 20-40 patients with hemophilia A and B. However Cuenca may host approximately 10,000 people with vWD because it affects both males and females. One plausible explanation for this lag is that the diagnostic suspicions of vWD had not been proven because of the lack of a special coagulation laboratory in Cuenca that would allow professionals to make a positive diagnosis.

This is the first clinical, demographic and socio-economic study of patients registered with hemophilia and of Cuenca women with possible vWD who are referred from dentists and physicians from public and private health centers. The instruments will be validated surveys and screening coagulation studies.

As far as we know Ecuador has not published studies on coagulopathies. Our aim is to initiate a process to eventually reach the goal to offer adequate diagnostic and therapeutic interventions to human victims of these diseases, within a program of service, teaching and research.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 81
• Est. completion date: June 2013
• Est. primary completion date: June 2013
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years to 50 Years

Eligibility

Inclusion Criteria:

• Subjects living at urban and rural parishes from Cuenca district.

• Subjects referred from professionals of public and private health services, who have filled the initial hemorrhage survey.

• Must be referred women who sign the informed consent form.

• Patients who answer the standardized bleeding score questionnaire (BS).

• Patients who answer the quality of life questionnaire (SF-12).

• Patients who answer the socio-economic survey.

• Patients who accept to provide a venous blood sample for the initial laboratory tests.

Exclusion Criteria:

• Women with organic or hormonal bleeding etiologies.

• Patients taking medicines which may affect the coagulation cascade or the number or function of platelets.

• Those women who otherwise do not comply with the inclusion criteria.

Study Design

N/A

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B
• Von Willebrand Disease
• Von Willebrand Diseases

Locations

Country	Name	City	State
Ecuador	UAzuay	Cuenca	Azuay
Ecuador	Universidad del Azuay (UDA)	Cuenca	Azuay

Sponsors (1)

Lead Sponsor	Collaborator
Universidad del Azuay

Country where clinical trial is conducted

Ecuador, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Bleeding Score (BS)	As an adequate bleeding history is fundamental, the Condensed MCMDM-1 Bleeding Questionnaire will be used. Its bleeding score (BS) has a sensitivity of 100%, specificity 87%, positive predictive value 0.2 and negative predictive value 1.0. There was a signifficant difference of BS between the subtypes of vWD: with type 3 >>type 2 >>type 1 vWD (Anova P <0.001).; AS the BS is an index of ordinal values, medians and the Mann-Whitney non-parametric ranksum test will be used, to study the bleeding score (BS) differences among subgroups of enrolled women.	1 week	No
Secondary	Associations among BS and female subgroup characteristics and initial laboratory tests.	As bleeding may tend to associate in subjects, i.e. epistaxis and menorrhagia, a logistic (log-linear) model will be used. As bleeding can also ocur independently, symptoms will be coded for an adjusted covariate analysis.; To study the relationships among the BS and the quantitative laboratory results, the BS will be divided in quintiles, and an adjusted multiple regression model will be used. Likelihood ratios (LR) for values of BS, with a prevalence of 5% and ROC curves will be applied. According to Tosetto et.al. laboratory data will be expressed in categorical and quantitative scales.	1 week	No