Abstract Number: PB1497
Meeting: ISTH 2020 Congress
Theme: Platelet Disorders and von Willebrand Disease » Platelet Function Disorders, Hereditary
Background: The ISTH Bleeding Assessment Tool (ISTH-BAT) and the Pediatric Bleeding Questionnaire (PBQ) were initially developed for persons with suspected von Willebrand disease. The diagnostic utility of these bleeding scores in patients with inherited platelet function disorders (IPFD) is still elusive.
Aims: To investigate the utility of the ISTH BAT and the PBQ in prediction of platelet function defects in children with excessive bleeding and suspicion for IPFD.
Methods: We enrolled 170 patients with bleeding complaints and without evidence of thrombocytopenia and coagulopathies. The ISTH-BAT and the PBQ scores were applied by a trained investigator prior to light transmission aggregometry (LTA) and platelet flow cytometry (PFC) with activation. A ROC-curve analysis was performed to evaluate whether the ISTH BAT and the PBQ scores could discriminate between patients with and without demonstrable defects in platelet function tests.
Results: Diagnosis of Glanzmann thrombasthenia (GT) was made in 21 patients. Other defects in platelet function tests were revealed in another 42 kids and there were no pathological findings in 107 patients. Both ISTH BAT and PBQ scores differs between GT patients and patients with other defects and between patients with other defects and group with no pathological findings (p< 0.05 in all pairwise comparisons). But difference between two last mentioned groups was less than 1 point (0,85 (CI 95% 0.19;1.50) for PBQ and 0,85 (CI 95% 0.18; 1.52) for ISTH BAT). AUC was 0.728 (CI 95% 0.647; 0.809) for PBQ and 0.730 (CI 95% 0.648; 0.812) for ISTH BAT for all patients. But after exclusion of GT patients AUC was only 0.610 (CI 95% 0.510; 0.711) for PBQ and 0.610 (CI 95% 0.508; 0.711) for ISTH BAT.
Conclusions: The ISTH-BAT and the PBQ questionnaires are reasonable tools for documenting of bleeding history. However, both scores are not predictive for non-severe platelet functional defects in patients with excessive bleeding.
| GT patients | Patients with other defects | Patients with normal tests | p-value (Kruskal-Wallis test) | |
| Number of patients (N) | 21 | 42 | 107 | – |
| M/F | 7/14 | 20/22 | 50/57 | – |
| Median age (IQR), year | 5 (1-10) | 7 (4-13) | 9 (5-13) | 0.006 |
| Median PBQ score (IQR) | 7 (5.5-9.5) | 3 (2-4) | 2 (2-4) | < 0.00001 |
| Median ISTH BAT score (IQR) | 8 (6.5 – 10.5) | 3 (2-4) | 2 (2-4) | <0.00001 |
[Bleeding scores in patients with GT, patients with other defects in platelet function tests and patients with no pathological findings.]
[ROC curve for presence of a defect in LTA and/or PFC and the bleeding scores.]
To cite this abstract in AMA style:
Fedorova D, Ignatova A, Polokhov D, Ponomarenko E, Seregina E, Poletaev A, Panteleev M, Zharkov P. Utility of the ISTH Bleeding Assessment Tool and the Pediatric Bleeding Questionnaire in predicting platelet function defects in pediatric patients with excessive bleeding [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/utility-of-the-isth-bleeding-assessment-tool-and-the-pediatric-bleeding-questionnaire-in-predicting-platelet-function-defects-in-pediatric-patients-with-excessive-bleeding/. Accessed October 1, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/utility-of-the-isth-bleeding-assessment-tool-and-the-pediatric-bleeding-questionnaire-in-predicting-platelet-function-defects-in-pediatric-patients-with-excessive-bleeding/