Generation and optimization of the self-administered pediatric bleeding questionnaire and its validation as a screening tool for von Willebrand disease

Discriminating children who have a bleeding disorder using their personal history remains difficult and several bleeding scores have been developed to discern children at increased probability for a bleeding disorder from those without. While most bleeding questionnaires have been expert-administered, this article presents an evaluation of a newly developed self-administered bleeding score (Self-PBQ, administered by caregivers or children).
This study was set up in three different phases: Phase 1: Translation of existing pediatric bleeding questionnaire items into lay language targeting reading level grade 4 and evaluation of the correlation with expert-administered questionnaires using known patients with von Willebrand disease type I (vWD I). Phase 2: Establishment of the normal range of the bleeding scores with the Self-PBQ in children with and without vWD I. Phase 3: Evaluation of the developed Self-PBQ for screening in first-time referrals (complaint being bleeding symptoms or family history) to the hematology clinic.
Phase I was performed on 38 patients with vWD I and without bleeding disorders (controls) and showed good correlation between the Self-PBQ and expert-administered bleeding scores after several rounds of revisions. Phase 2 using 56 children with and without vWD I showed that all tested controls had bleeding scores of 0-2 which led to the definition of a bleeding score cutoff of >2 being abnormal. Phase 3 in 155 children referred to a hematology clinic showed excellent correlation of the Self-PBQ and the expert-administered PBQ. Of 23 patients with VWD I, 5 were assigned a negative BS, all of them had a positive family history of vWD. The negative predictive value was 91%. Median time for completion was 10 minutes.
The same limitations apply to all existing bleeding scores, they perform moderately well to rule out minor bleeding disorders (vWD I). Particularly young children might be missed as they haven't faced major hemostatic challenges. So far, this bleeding score has only been evaluated for vWD I, it would be highly desirable to test it in other bleeding disorders. Family history seems needed to adequately assess individual patients' risks for a bleeding disorder which is not part of the Self-PBQ.
Pediatric bleeding scores are a valuable tool to assess the pretest probability in patients referred to a hematology clinic for a possible bleeding disorder. This self-administered tool (Self-PBQ) performs equally well compared to expert-administered questionnaires and is a useful tool to assess referrals before consultation to a hematologist.
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