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

Abstract

The Self-PBQ generates comparable BSs to the expert-administered PBQ and is a reliable, reasonably sensitive screening tool to incorporate into the assessment of children presenting to a hematologist for the investigation of an inherited bleeding disorder.

Validation of the school age self‐administered pediatric bleeding questionnaire (Self‐PBQ) in children aged 8–12 years

“…Two populations were recruited from CHEO: (1) children with a confirmed diagnosis of type 1 VWD and (2) normal children without bleeding disorders. Patients with type 1 VWD were defined as those with a VWF antigen (VWF:Ag) and VWF ristocetin cofactor/activity (VWF:RCo/Act) between 0.05 and 0.50 U/mL on two occasions, a RCo:Ag/Act ratio > 0.60, and normal VWF multimers, based on laboratory criteria from previous studies . Normal controls were either unaffected siblings of the patients with normal VWF levels or children with unrelated disorders.…”

“…The Self‐PBQ demonstrated a sensitivity of 78%, a specificity of 37%, a positive predictive value of 0.18, and a negative predictive value of 0.91 for VWD. Furthermore, the Self‐PBQ had a high level of agreement with the expert‐administered PBQ, with an intraclass correlation (ICC) of 0.917 . However, only children above the age of 12 completed the Self‐PBQ by themselves while those under 12 had a parent fill out the questionnaire on their behalf.…”

“…Although the Self‐BAT was validated for adult patients only, Casey et al subsequently undertook the validation of a self‐administered bleeding questionnaire for use in a pediatric population. The self‐administered PBQ (Self‐PBQ) was generated by combining the expert‐administered PBQ and the ISTH‐BAT, then translating the questionnaire into terms that can be understood by children and their parents . A prospective multicenter study was carried out to validate the Self‐PBQ as a screening tool for VWD in children presenting to the hematology clinic for investigation of a bleeding disorder.…”

Reply to comment on: Generation and optimization of the self‐administered pediatric bleeding questionnaire and its validation as a screening tool for von Willebrand disease

“…In their letter, Attard and colleagues express concern with the Self‐PBQ's use of medical nomenclature, namely the terms “intravenous (IV)” and “desmopressin—DDAVP, octostim or stimate.” They are concerned that these terms are unlikely to be understood by the layperson, decreasing the tool's effectiveness. Though we would argue that “IV” is common terminology in media and culture, fundamentally, its use does not significantly impact the properties of the tool: as we demonstrated, the Self‐PBQ was sufficiently sensitive to identify the majority of von Willebrand disease (VWD) cases . Indeed, we are currently studying administration of the Self‐PBQ in participants as young as 8 years, and have observed excellent agreement of children's scores with their parents' scores (manuscript in preparation).…”

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

“…We would like to congratulate Casey et al. on the development of their self‐administrable pediatric bleeding questionnaire (Self‐PBQ) as a screening tool for von Willebrand disease . To date, similar screening tools have required expert administration, generally in a clinical setting.…”