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

Abstract

The diagnosis of mild bleeding disorders is not easy as most of the "healthy" individuals also report bleeding symptoms. In order to get a precise bleeding history, Pediatric Bleeding Questionnaire (PBQ) has been developed. In our study, Turkish children diagnosed with Von Willebrand disease (VWD), platelet function defect (PFD), and healthy children without any symptoms (control group 1) and healthy children with symptoms but found hemostatically normal (control group 2) were analyzed with PBQ. The cut off level for "positive bleeding score" was found to be ≥2 (area under the curve [AUC]: 0.785, 95% confidence interval [CI]: 0.718-0.852). The sensitivity, specificity, positive predictive value, and negative predictive value of PBQ to define VWD versus control group 1 was 100%, 97.4%, 96.4%, and 100%; VWD versus control group 2 was 100%, 53.1%, 64.3%, and 100%; PFD versus control group 1 was 93.3%, 53.1%, 73.7%, and 85%; and PFD versus control group 2 was 93.3%, 53.1%, 73.7%, and 85%, respectively.

Weak correlation of bleeding scores to platelet electron microscopy: A retrospective chart review of pediatric patients with delta‐storage pool disorder

“…Previous studies have established BAT use in pediatric patients when screening for mild bleeding disorders, showing promise when focusing on patients with VWD. 21,23,24,27 However, those studies which used the BAT in the pediatric population included only a few -SPD patients. 9,23,25 The bleeding score average for the analyzed patients was similar to the previously published average for -SPD patients.…”

“…Once the BAT is administered, the clinician calculates the sum of the individual bleeding categories to establish the total score with a possible range from 0 to 48; a score ≥2 indicates a positive screen. A normal screen (score ≤1) is estimated to miss 1 out of 500 pediatric patients referred, despite children having less lifetime hemostatic challenges …”

“…A normal screen (score ≤1) is estimated to miss 1 out of 500 pediatric patients referred, despite children having less lifetime hemostatic challenges. 21…”

“…22 Although earlier studies questioned the accuracy and negative predictive value of the BAT in younger children since they would have lower scores due to fewer hemostatic challenges, 23 when the ISTH-BAT was utilized on children with bleeding symptoms, the observed sensitivity, specificity, and negative predictive value showed effectiveness as a screening tool. 21,[24][25][26][27][28] In children, a high BAT score strongly correlated to abnormal light transmission aggregometry. 25 When applied to alpha and -SPD patients, researchers observed an average BAT score of 5.…”

“…20 Utility evaluation studies have since shown the BAT to be a powerful screening tool for patients with Von Willebrand disease (VWD) or platelet function disorders. 21 Recent data for adults with VWD suggests that appropriate cutoff values for adult males and females are higher than the cutoff value for children. 22 Although earlier studies questioned the accuracy and negative predictive value of the BAT in younger children since they would have lower scores due to fewer hemostatic challenges, 23 when the ISTH-BAT was utilized on children with bleeding symptoms, the observed sensitivity, specificity, and negative predictive value showed effectiveness as a screening tool.…”