Prospective evaluation of a pediatric bleeding questionnaire and the ISTH bleeding assessment tool in children and parents in routine clinical practice

Abstract

Background: Diagnosing mild bleeding disorders (BDs) in children is difficult. Bleeding scores (BSs) have been proposed for obtaining standardized quantitative histories.

Objectives: To compare the Canadian pediatric bleeding questionnaire (PBQ) with the new ISTH bleeding assessment tool (ISTH BAT) for the determination of BS in a routine pediatric outpatient setting.

Methods: One hundred children with a suspected BD were enrolled in this cross-sectional study. Bleeding scores were calculated for all children and their natural parents. For all children, extensive laboratory investigations were performed.

Results: Based on laboratory tests, 56 children were diagnosed as having no BD, 11 were diagnosed with possible VWD, 12 with VWD 1, 11 with VWD 2, five with possible platelet defects, and five with mild factor deficiencies. Both questionnaires were able to discriminate between no BD and VWD (P = 0.0001), but the area under the receiver characteristics curve to detect any mild BD was only 0.76. Despite the inherited nature of the BD, a family score did not increase the ability to discriminate between no BD and VWD (P = 0.2052). There was no significant difference between the two tools used (P = 0.3253) or simple qualitative criteria, such as yes/no questions regarding bleeding (P = 0.3477).

Conclusions: The two tools translated into German did not differ substantially. Both were able to discriminate between no BD and a possible BD with acceptable accuracy. A BS of < 2 makes a BD unlikely. Simple qualitative criteria were similar; however, to allow comparison of studies and follow-up in patients over time, we recommend the ISTH BAT.