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Generation and optimization of the self-administered pediatric bleeding questionnaire and its validation as a
Lara J Casey1, Angie Tuttle1, Julie Grabell1
1Queen's University, Kingston, Canada.
Insights
A new pediatric bleeding questionnaire (Self-PBQ) reliably screens children for von Willebrand disease (VWD). This tool is a sensitive and valuable addition for hematologists investigating inherited bleeding disorders.
Area of Science:
- Pediatric Hematology
- Hemostasis and Thrombosis
- Clinical Diagnostics
Background:
- Von Willebrand disease (VWD) is a common inherited bleeding disorder.
- Accurate screening tools are crucial for early diagnosis in children.
- Existing tools may require expert administration, limiting accessibility.
Purpose of the Study:
- To develop and validate a self-administered pediatric bleeding questionnaire (Self-PBQ).
- To establish the Self-PBQ as a reliable screening tool for VWD in pediatric hematology referrals.
- To ensure the questionnaire is accessible with a grade 4 reading level.
Main Methods:
- Combined validated expert-administered PBQ and ISTH-BAT into the Self-PBQ.
- Translated medical terms to lay language (grade 4 reading level).
- Validated the Self-PBQ against expert-administered PBQ and ISTH-BAT, establishing normal bleeding score ranges and assessing its screening performance.
Main Results:
- The Self-PBQ demonstrated excellent agreement with the expert-administered PBQ (ICC=0.917).
- Normal Self-PBQ bleeding score ranges were determined (-1 to 2 or 0 to 2).
- A positive Self-PBQ score (≥3) showed 78% sensitivity and 91% negative predictive value for VWD.
Conclusions:
- The Self-PBQ is a reliable tool generating comparable bleeding scores to expert-administered questionnaires.
- It serves as a reasonably sensitive screening tool for inherited bleeding disorders in children.
- The Self-PBQ can be effectively incorporated into the initial assessment of pediatric patients presenting to hematologists.
Objective:
Our objective was to generate, optimize, and validate a self-administered pediatric bleeding questionnaire (Self-PBQ) as a screening tool for von Willebrand disease (VWD) in children referred to the hematology clinic for the first time.
Study Design:
The Self-PBQ was generated by combining the validated expert-administered PBQ and the International Society on Thrombosis and Hemostasis (ISTH) bleeding assessment tool (BAT). Medical terminology was translated into lay language requiring a grade 4 reading level. In Phase 1, the Self-PBQ was optimized and the level of agreement between the Self-PBQ and the expert-administered PBQ was determined. Phase 2 established the normal range of bleeding scores (BSs) of the Self-PBQ. Phase 3 examined the Self-PBQ as a screening tool for first-time referrals to the hematology clinic.
Results:
The Self-PBQ is a reliable surrogate for the expert-administered PBQ with an excellent intraclass correlation (ICC) of 0.917. The Self-PBQ was scored with the PBQ and the ISTH-BAT scoring systems, for which its normal BS ranges are -1 to 2 or 0 to 2, respectively. A positive Self-PBQ BS (≥3) had a sensitivity of 78%, a specificity of 37%, a positive predictive value of 0.18, and a negative predictive value of 0.91 for identifying VWD in children being investigated by a hematologist for a bleeding disorder.
Conclusion:
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.
