Prospective Comparison of the Pediatric Bleeding Questionnaire (PBQ) and the International Society On Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) in Children Referred to a Tertiary-Care Pediatric Centre.

Abstract 2229 Mucocutaneous bleeding symptoms, such as epistaxis and bruising, are frequent complaints in childhood; a detailed bleeding history is a crucial initial step in determining whether a child presenting with such symptoms has an underlying bleeding disorder. A Pediatric Bleeding Questionnaire (PBQ), based on the MCMDM-1 VWD Bleeding Questionnaire, incorporates 6 pediatric-specific bleeding symptoms (see below) to quantify bleeding severity in children. Bleeding symptoms are scored in a −1 to +4 range, with a −1 score assigned for tooth extraction or surgery if bleeding did not occur in at least 2 procedures, and are summed for all symptoms. An overall PBQ score of ≥2 predicts a diagnosis of von Willebrand disease (VWD). The PBQ has also been used to quantify the severity of bleeding symptoms in children with VWD or platelet function disorders (Bowman et al, J Thromb Haemost 2009;7:1418; Biss et al, J Thromb Haemost 2010;8:950; Biss et al, J Thromb Haemost 2010;8:1416). The ISTH has developed a Bleeding Assessment Tool (ISTH-BAT) to standardize the reporting of bleeding symptoms in adult and pediatric populations; bleeding symptoms are scored in a 0 to +4 range, and are summed (Rodeghiero et al, J Thromb Haemost 2010;8:2063). Criteria for scoring of each symptom are similar between the two questionnaires, but not identical. Here, we have performed a detailed comparison between PBQ and ISTH-BAT scores in a prospective study of children with mucocutaneous bleeding and/or a family history of VWD or a platelet function disorder referred to our tertiary-care pediatric bleeding disorders clinic. To date, we have enrolled 75 subjects, with a mean age of 9.9 yrs (range: 0.5–17.8 yrs), of whom 36 are males. The median overall PBQ score of these children was 3 (range: 0–12), as was the median overall ISTH-BAT score (range: 0–13). In 37/75 children (49%), the overall PBQ score was identical to the overall ISTH-BAT score. In the majority of these children (34/37; 92%), the individual scores for each symptom were identical. However, in 3 children, there were differences in the individual scores that balanced out, resulting in identical overall scores. For 38/75 children (51%), the overall PBQ and ISTH-BAT scores were different. In the majority of these children (33/38; 87%), the difference between the scores was only 1, with the ISTH-BAT being lower in 19/38 children, and higher in 14/38 children. In 2/38 children, the overall ISTH-BAT was lower by 2, in 2/38, higher by 2 and in 1/38, higher by 3. A lower overall ISTH-BAT score was mainly due to a lower score for cutaneous bleeding symptoms (14/21 children). A higher overall ISTH-BAT score was mainly due to a −1 PBQ score for a child who did not bleed on at least 2 tooth extractions or surgeries (observed in 11/17 children) and/or a higher ISTH-BAT score for menorrhagia (observed in 6/17 children, specifically, 6/9 postmenarchal adolescent females). 10/75 (13%) children had a normal overall PBQ score of 0 or 1 (median: 1). The median overall ISTH-BAT score in these children was also 1 (range: 0–3), but 5 children had a score of 2 or 3. In the remaining 65/75 children (87%) with a positive, abnormal PBQ score, the median score was 3 (range: 2–12), as was the median overall ISTH-BAT score (range: 2–13). In contrast with previous prospective studies using the PBQ/ISTH-BAT in which pediatric-specific symptoms were not observed (Bowman et al, J Thromb Haemost 2009;7:1418; Bidlingmaier et al, J Thromb Haemost 2012;10:1335), 12/75 children (16%) received scores for macroscopic hematuria, post-circumcision bleeding, cephalohematoma, umbilical stump bleeding, post-venipuncture bleeding, or conjunctival hemorrhage. In summary, in this prospective study, we have observed similar, but not identical, overall PBQ and ISTH-BAT scores and the occurrence of pediatric-specific bleeding symptoms in children referred to a tertiary-care bleeding disorders clinic with mucocutaneous bleeding and/or a family history of VWD or platelet dysfunction. Thus, the inclusion of pediatric-specific bleeding symptoms in the standardized questionnaires is useful, and if the ISTH-BAT is to be adopted for general use to aid in the evaluation of whether a child has an underlying bleeding disorder, it will be essential to determine the cut-off for an abnormal ISTH-BAT bleeding score in children <18 yrs of age. Disclosures: James: CSL-Behring, Baxter, Bayer: Honoraria, Research Funding.