Abstract Number: PB0900
Meeting: ISTH 2021 Congress
Theme: Platelet Disorders, von Willebrand Disease and Thrombotic Microangiopathies » Platelet Function Disorders, Hereditary
Background: GT is one of the most severe platelet functional disorders. Platelet transfusion and rFVIIa are available therapeutic options for severe bleeding. Nevertheless, there is no effective conventional prophylaxis of bleeding in GT patients nowadays.
Aims: To assess bleeding phenotype in pediatric GT patients and compare it with phenotype of other hereditary hemorrhagic disorders.
Methods: We used ISTH Bleeding Assessment Tool (ISTH BAT) and Pediatric Bleeding Questionnaire (PBQ) scales for assessment and documenting of hemorrhagic symptoms in 25 children (8 boys and 17 girls) with GT. We compared bleeding scores of GT patients with two other groups. The first group included children with von Willebrand disease (overall 35 patients, 19 – type 1, 13 – type 2, 3 – type 3), and the second group included 38 children with other platelet functional disorders.
Results: Median age at admission to our tertiary hospital was 5 years (IQR 1.5;7) in patients with GT. All patients had bleeding in more than one site.
Bleeding symptoms in pediatric GT patients.
All 8 school-age patients reported missing school because of bleeding episodes. Median age of onset of hemorrhagic symptoms was 1 month (IQR 0;6). Twenty-three (92%) patients required emergency medical care for management of bleeding episodes. Twelve (48%) patients received blood transfusion at least once. Median bleeding score was 8 (IQR 6.5;11.5) according to the ISTH BAT and 7 (IQR 5.5;10.5) according to the PBQ. Scores on both scales were significantly higher in GT patients compared to the other two groups (p<0.05 in pair-wise comparisons).
| Parameters | GT (n=25) | Other IPFD ( n=38) |
vWD (n=35) | р-value |
| Median age at admission (IQR), months | 64 (20;88) | 77 (39;186,25) | 72 (36;120) | .13 |
| Median PBQ score (IQR) | 7 (5,5;10,5) | 3 (2;5) | 4 (2;5,25) | < .00001. |
| Min/max PBQ score | 2/14 | 1/16 | 1/12 | – |
| Median ISTH BAT score (IQR) | 8 (6,5;11,5) | 3 (2;5) | 4 (2;5,25) | < .00001 |
| Min/max ISTH BAT score | 3/15 | 1/17 | 4 (2;5,25) |
– |
| >1 bleeding sites, n (%) | 25 (100) | 24 (63) | 25 (71) | .0007 |
| Iron deficiency, n (%) | 17 (68) | 14 (37) | 15 (43) | .04 |
| Emergency medical care, n (%) | 23 (92) | 17 (45) | 14 (40) | .00003 |
| Blood transfusions or replacement therapy, n (%) | 13 (52) | 8 (21) | 12 (34) | .03 |
Bleeding severity in patients with GT, other inherited functional platelet disorders (IFPD) and von Willebrand disease (vWD)
We could not demonstrate significant correlation between age and ISTH BAT and PBQ scores (р=0.58 and 0.4, accordingly) in GT patients. Probably, correlation was not significant because of absence of adolescent girls in our group.
Conclusions: Patients with GT demonstrate severe bleeding phenotype since first months of life. Majority of pediatric GT patients need emergency medical care for bleeding management.
To cite this abstract in AMA style:
Fedorova D, Seregina E, Poletaev A, Pshonkin A, Zharkov P. Hemorrhagic Phenotype in Pediatric Patients with Glanzmann Thrombasthenia (GT) [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/hemorrhagic-phenotype-in-pediatric-patients-with-glanzmann-thrombasthenia-gt/. Accessed August 16, 2022.« Back to ISTH 2021 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/hemorrhagic-phenotype-in-pediatric-patients-with-glanzmann-thrombasthenia-gt/