Evaluation of Pediatric Bleeding Questionnaire in Turkish Children With Von Willebrand Disease and Platelet Function Disorders

Area of Science:

• Pediatric Hematology
• Clinical Diagnostics
• Medical Questionnaires

Background:

• Diagnosing mild bleeding disorders in children presents challenges due to overlapping symptoms in healthy individuals.
• The Pediatric Bleeding Questionnaire (PBQ) was developed to improve the precision of bleeding history assessment.

Purpose of the Study:

• To evaluate the efficacy of the PBQ in differentiating between children with Von Willebrand disease (VWD), platelet function defects (PFD), and healthy controls.
• To establish a reliable cutoff score for the PBQ to identify children with bleeding disorders.

Main Methods:

• The study involved Turkish children diagnosed with VWD, PFD, and two control groups: healthy children without symptoms and healthy children with symptoms but normal hemostasis.
• The Pediatric Bleeding Questionnaire (PBQ) was administered to all participants.
• Statistical analysis was performed to determine the diagnostic accuracy, including sensitivity, specificity, and predictive values, with an Area Under the Curve (AUC) calculation.

Main Results:

• A cutoff bleeding score of ≥2 was identified as indicative of a positive diagnosis (AUC: 0.785).
• PBQ demonstrated high sensitivity (100%) and negative predictive value (100%) for VWD against control group 1.
• For PFD, PBQ showed 93.3% sensitivity and 85% negative predictive value against control group 1.

Conclusions:

• The PBQ is a valuable tool for diagnosing mild bleeding disorders in children, particularly VWD.
• The established cutoff score of ≥2 provides a reliable threshold for identifying potential bleeding conditions.
• Further validation may be beneficial for differentiating PFD, especially against control group 2.