Von Willebrand's Diseased Children, Bleeding Questionnaire is DoneNCT04849338
The Role of Standardized Bleeding Score in Diagnosis of Pediatric Von Willebrand's Disease
An observational study of Von Willebrand's Diseased Children, Bleeding Questionnaire is Done, run by Sohag University at 1 site in Egypt.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 week to 18 years
- Conditions the study is for
- Von Willebrand's Diseased Children, Bleeding Questionnaire is Done
- What is being tested?
- Bleeding score ( questionnaire )
- How many people
- 50 participants
- Where
- 1 siteEgypt
- Study dates
- 15 Apr 2021to 15 May 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 week to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: children, aged 1 week-18 years, referred to Sohag university hospital , von Willebrand's diseased
Who may be able to join
- 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.
- 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.
Who cannot join
- Patients older than 18 years.
- Other inherited or acquired bleeding disorders.
- Patients on regular antiplatelets or anticoagulants.
- Patients older than 18 years.
- Other inherited or acquired bleeding disorders.
- Patients on regular antiplatelets or anticoagulants.
Trial locations and contacts
1 site in 1 country.
About this study
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
Read the 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 .
- Being tested
- Bleeding score ( questionnaire )
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bleeding score ( questionnaire )Diagnostic test
Bleeding score is done for children diagnosed as von Willebrand's disease .
What is being measured
The primary outcome is the main question the study is designed to answer.
Use bleeding score as diagnostic tool of von Willebrand's disease
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
Measured: Within one year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2021
- Study start15 Apr 2021 (estimated)
- Primary completion15 May 2022 (estimated)
- Study completion15 May 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand's Diseased Children, Bleeding Questionnaire is DoneStudies of the same condition, open and under way.
- More trials run by Sohag UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sohag University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Marina Kenz Basta, Sohag University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04849338
- Sponsor's protocol ID
- Soh-Med-21-04-09
- Status last verified
- Apr 2021
- First submitted
- 14 Apr 2021
- Last updated
- 19 Apr 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Role of Standardized Bleeding Score in Diagnosis of Pediatric Von Willebrand's Disease
- ClinicalTrials.gov ID
- NCT04849338
- Acronym / sponsor ID
- Soh-Med-21-04-09
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Sohag UniversityUniversity, hospital or other
- Study start
- 15 Apr 2021 (estimated)
- Primary completion
- 15 May 2022 (estimated)
- Study completion
- 15 May 2022 (estimated)
- First posted
- 19 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 1 week to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- children, aged 1 week-18 years, referred to Sohag university hospital , von Willebrand's diseased
Key inclusion criteria (4 listed)
- 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.
- 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.
Key exclusion criteria (6 listed)
- Patients older than 18 years.
- Other inherited or acquired bleeding disorders.
- Patients on regular antiplatelets or anticoagulants.
- Patients older than 18 years.
- Other inherited or acquired bleeding disorders.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bleeding score ( questionnaire )Bleeding score is done for children diagnosed as von Willebrand's disease . | Diagnostic test | – |
Primary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2021.
- Individual participant dataSharing planned: Yes
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand's Diseased Children, Bleeding Questionnaire is Done, run by Sohag University at 1 site in Egypt.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Marina Kenz Basta (01027728754, marena011143@med.sohag.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Use bleeding score as diagnostic tool of von Willebrand's disease (time frame: Within one year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Apr 2021.