Lara J Casey1, Angie Tuttle1, Julie Grabell1, Wilma Hopman1, Paul C Moorehead2,3, Victor S Blanchette4, John K Wu5, MacGregor Steele6, Robert J Klaassen7, Mariana Silva1, Margaret L Rand8,9, Paula D James1. 1. Queen's University, Kingston, Canada. 2. Janeway Children's Health and Rehabilitation Centre, St. John's, Canada. 3. Discipline of Pediatrics, Faculty of Medicine, Memorial University, St. John's, Canada. 4. Division of Hematology/Oncology, Department of Pediatrics, University of Toronto, Hospital for Sick Children, Toronto, Canada. 5. British Columbia Children's Hospital, Vancouver, Canada. 6. Alberta Children's Hospital, Calgary, Canada. 7. Children's Hospital of Eastern Ontario, Ottawa, Canada. 8. Division of Hematology/Oncology and Program in Experimental Medicine, The Hospital for Sick Children. 9. Departments of Laboratory Medicine & Pathobiology, Biochemistry and Pediatrics, University of Toronto, Toronto, Canada.
Abstract
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.
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.
Authors: Jordan Spradbrow; Sasha Letourneau; Julie Grabell; Yupu Liang; James Riddel; Wilma Hopman; Victor S Blanchette; Margaret L Rand; Barry S Coller; Andrew D Paterson; Paula D James Journal: Res Pract Thromb Haemost Date: 2019-10-30
Authors: Eva Stokhuijzen; Margaret L Rand; Marjon H Cnossen; Tina T Biss; Paula D James; Monique H Suijker; Marjolein Peters; Johanna H van der Lee; Bram Peters; Alexander B Meijer; Victor S Blanchette; Karin Fijnvandraat Journal: BMJ Open Date: 2018-05-03 Impact factor: 2.692