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

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: Recruiting
• Conditions: Von Willebrand's Diseased Children, Bleeding Questionnaire is Done
• Intervention / Treatment: Diagnostic test: Bleeding score ( questionnaire )

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: Observational
• Enrollment (Anticipated): 50

Contacts and Locations

• Study Contact: Name: Marina Kenz Basta; Phone Number: 01027728754; Email: marena011143@med.sohag.edu.eg

Study Locations

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

Participation Criteria

Eligibility Criteria

• 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

How is the study designed?

Design Details

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Within one year

Collaborators and Investigators

• Sponsor: Sohag University

Study record dates

Study Major Dates

• Study Start (Anticipated): April 15, 2021
• Primary Completion (Anticipated): May 15, 2022
• Study Completion (Anticipated): May 15, 2022

Study Registration Dates

• First Submitted: April 14, 2021
• First Submitted That Met QC Criteria: April 14, 2021
• First Posted (Actual): April 19, 2021

Study Record Updates

• Last Update Posted (Actual): April 19, 2021
• Last Update Submitted That Met QC Criteria: April 14, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Hemorrhage; Von Willebrand Diseases
• Other Study ID 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