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Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador (VWD)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Jaime M. Moreno A., M.D., M.S., F.A.C.P., Universidad del Azuay
ClinicalTrials.gov Identifier:
NCT01589848
First received: April 30, 2012
Last updated: July 2, 2013
Last verified: July 2013

April 30, 2012
July 2, 2013
March 2013
June 2013   (final data collection date for primary outcome measure)
Bleeding Score (BS) [ Time Frame: 1 week ] [ Designated as safety issue: No ]

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.

Same as current
Complete list of historical versions of study NCT01589848 on ClinicalTrials.gov Archive Site
Associations among BS and female subgroup characteristics and initial laboratory tests. [ Time Frame: 1 week ] [ Designated as safety issue: No ]

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.

Same as current
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Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador
Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador
  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?

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.

Observational
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Retention:   Samples Without DNA
Description:

Blood samples

Non-Probability Sample

Target population: all women from Cuenca district with a hemorrhage history.

Accessible population: Those women referred from professionals with an initial hemorrhage history, who comply with the inclusion criteria and who sign the informed consert form.

Sample population: First comers referred with a hemorrhage history until fulfilling the projected sample size. This non-probabilistic purposive sample will be used because it has been reported that it could represent the target population.

*2010 Ecuadorian census: 131.163 women ages 18 to 50 years, at Cuenca district.

  • Von Willebrand Disease
  • Hemophilia A
  • Hemophilia B
Not Provided
von Willebrand women
Referred women who may have von Willebrand Disease
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
81
June 2013
June 2013   (final data collection date for primary outcome measure)

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.
Female
18 Years to 50 Years
No
Contact information is only displayed when the study is recruiting subjects
Ecuador
 
NCT01589848
001, UAzuay
Yes
Jaime M. Moreno A., M.D., M.S., F.A.C.P., Universidad del Azuay
Universidad del Azuay
Not Provided
Principal Investigator: Jaime M. Moreno A., M.D., M.S. Universidad del Azuay (UAzuay)
Study Chair: Jacinto Guillen G., Eng., M.S. Universidad del Azuay (UAzuay)
Study Director: Rodrigo Cueva M., Econ., M.S. Universidad del Azuay (UAzuay)
Universidad del Azuay
July 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP