The Role of Standardized Bleeding Score in Diagnosis of Pediatric Von Willebrand's Disease

April 14, 2021 updated by: Marina Kenz Basta, Sohag University
Von Willebrand's disease (VWD) is the most common inherited bleeding disorder. It arises from a deficiency in the quality or quantity of von Willebrand factor (VWF), a multimeric protein that is required for platelet adhesion. Typical patients present with mucocutaneous bleeding symptoms because of reduced circulating von Willebrand factor (VWF) In this study we will use the ISTH/SSC BLEEDING assessment tool as a standardized questionnaire and a proposal for a new bleeding score for inherited bleeding disorders

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Von Willebrand's disease (VWD) is the most common inherited bleeding disorder. It arises from a deficiency in the quality or quantity of von Willebrand factor (VWF), a multimeric protein that is required for platelet adhesion. Typical patients present with mucocutaneous bleeding symptoms because of reduced circulating von Willebrand factor (VWF) . A predictive bleeding score could reveal individuals who may benefit from repetitive testing and those for whom repetitive testing is unlikely to be of benefit . In 2005, Rodeghiero, et al. developed a standardized questionnaire for the evaluation of haemorrhagic symptoms, referred to hereafter as the Vicenza score. Their retrospective analysis found that >3 haemorrhagic symptoms or bleeding scores of 3 in men and 5 in women was very specific (98.6%) for VWD, although less sensitive (69.1%)]. These investigators later reported in a larger population that higher bleeding scores were associated with increasing likelihood of VWD and bleeding after surgery or tooth extraction. Neither of these studies prospectively surveyed paediatric populations. As a result of their shorter life experience, children have fewer exposures to bleeding challenges such as dental extractions, surgeries, menarche and childbirth. In 2009, Bowman, et al. administered a modified Vicenza bleeding score to a naive primary care paediatric population and determined a bleeding score ≥2 was abnormal. Using this definition, their bleeding score had a high negative predictive value (0.99) and could accurately distinguish between children with and without VWD. In this study we will use the ISTH/SSC BLEEDING assessment tool as a standardized questionnaire and a proposal for a new bleeding score for inherited bleeding disorders. For each specific bleeding symptom, the ISTH/SSC joint working group proposed minimal criteria in order to classify a symptom as significant and thus receive a score of 1 or more. Symptoms included in this questionnaire are epistaxis , cutaneous bleeding , minor cutaneous wound , oral cavity bleeding , hematemesis , melena and hematochezia , hematuria , tooth extraction , surgical bleeding , menorrhagia , postpartum bleeding , muscle hematomas or hemoarthrosis and CNS bleeding .

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Egypt
      • Sohag, Egypt, 82768
        • Recruiting
        • Sohag University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 week to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

children, aged 1 week-18 years, referred to Sohag university hospital , von Willebrand's diseased

Description

Inclusion Criteria:

  • This study included children, aged 1 week-18 years, referred to Sohag university hospital for evaluation of bleeding symptoms, family history of a bleeding disorder and/or abnormal coagulation studies.
  • Bleeding questionnaire is done during patients' clinic visit.

Exclusion Criteria:

  1. Patients older than 18 years.
  2. Other inherited or acquired bleeding disorders.
  3. Patients on regular antiplatelets or anticoagulants.

Inclusion Criteria:

  • This study included children, aged 1 week-18 years, referred to Sohag university hospital for evaluation of bleeding symptoms, family history of a bleeding disorder and/or abnormal coagulation studies.
  • Bleeding questionnaire is done during patients' clinic visit.

Exclusion Criteria:

  1. Patients older than 18 years.
  2. Other inherited or acquired bleeding disorders.
  3. Patients on regular antiplatelets or anticoagulants.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Cross-Sectional

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Use bleeding score as diagnostic tool of von Willebrand's disease
Time Frame: Within one year
The aim of the present study is to assess the diagnostic power of the bleeding score in identifying VWD in a prospective cohort of patients referred to a paediatric haematology clinic for evaluation of a bleeding disorder , so we can predict the next bleeding episode and minimize the complications
Within one year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sohag University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 15, 2021

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 15, 2022

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 15, 2022

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 14, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 14, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 19, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 19, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 14, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • Soh-Med-21-04-09

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

IPD Sharing Supporting Information Type

  • Study Protocol

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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