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

NCT ID: NCT04849338

Last Updated: 2021-04-19

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 50 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-04-15
• Study Completion Date: 2022-05-15

Brief 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. 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 .

Conditions

Von Willebrand's Diseased Children, Bleeding Questionnaire is Done

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: CROSS_SECTIONAL

Interventions

Bleeding score ( questionnaire )

Bleeding score is done for children diagnosed as von Willebrand's disease .

Intervention Type: DIAGNOSTIC_TEST

Eligibility Criteria

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.

• 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.

1. Patients older than 18 years.

2. Other inherited or acquired bleeding disorders.

3. Patients on regular antiplatelets or anticoagulants.

• Minimum Eligible Age: 1 Week
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sohag University

OTHER

Sponsor Role: lead

Responsible Party

Marina Kenz Basta

resident doctor at pediatric department sohag university hospital

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Sohag University

Sohag, , Egypt

Site Status: RECRUITING

Countries

Egypt

Central Contacts

Marina Kenz Basta

Role: CONTACT

marena011143@med.sohag.edu.eg

01027728754

Other Identifiers

Soh-Med-21-04-09

Identifier Type: -

Identifier Source: org_study_id