PB0552 - Phenotypic Characterization of Moderate and Mild Type Hemophilia A by Thrombin Generation Assay

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Yeu-Chin Chen, MD

Associate Professor/Doctor, Director of hemophilia care & research center
Division of Hematology/Oncology, Tri-Service General Hospital, Defense Medical Center
Taipei, Taipei, Taiwan (Republic of China)

Co-Author(s)

CC

Chia-Yau Chang

Assistant professor, Director of hemophilia center
1. School of Medicine, Graduate Institute of Clinical Medicine, Taipei Medical University, Taipei, Taiwan 2Hemophilia care & research center, Division of Pediatric Hematology/Oncology, Taipei Medical University Hospital, Taipei, Taiwan
Taipei, Taipei, Taiwan (Republic of China)
TL

Tsung-Ying Li

Associate professor
Department of Physical Medicine and Rehabilitation, Tri-Service General Hospital, National Defense Medical Center, Taipei, Taiwan
Taipei, Taipei, Taiwan (Republic of China)
SL

Shiue-Wei Lai

Assistant professor, attending physician
Hemophilia care & research center, Division of Hematology/Oncology, Tri-Service General Hospital, National Defense Medical Center
Taipei, Taipei, Taiwan (Republic of China)
DW

Der-Shiun Wang

Lecturer, attending physician
1.Hemophilia care & research center, Tri-Service General Hospital, National Defense Medical Center 2. Department of Pediatrics, Tri-Service General Hospital, National Defense Medical Center, Taipei, Taiwan
Taipei, Taipei, Taiwan (Republic of China)
SH

Shu-Hsia Hu

Research associate
Hemophilia care & research center, Division of Hematology/Oncology, Tri-Service General Hospital, National Defense Medical Center
Taipei, Taipei, Taiwan (Republic of China)

Background: The thrombin generation assay (TGA), a global hemostasis test, has been reported that its endogenous thrombin potential (ETP) may have good correlation with bleeding phenotype in severe type hemophilia A (HA) patients. However, its application in non-severe type HA is less reported.

Aims: The aims of this study were (1) to characterize the non-severe type patients by the parameters of TGA, and (2) to explore whether TGA can be used for distinguish the bleeding phenotype between mild and moderate type HA patients.

Methods: There were 31 consecutive male non-severe type HA patients including 21 mild and 10 moderate type without prophylaxis therapy, who were enrolled from 2022/1/13~2023/7/21. The median patients’ age was 37 years old, ranged from 8~85. Patients’ clinical informations including age, FVIII:C, annual joints bleeding rate and annual bleeding rate and condition of hemophilic arthropathy were collected. Hemophilic arthropathy was assessed by three assessment tools including plain X-ray, HJHS and ultrasonography by HEAD-US score. Each patient's peripheral platelet poor plasma was collected and frozen for the TGA.

Results: Of the 31 patients, the median value of lag time, velocity index, peak time, peak thrombin and ETP were all significantly different compared with those data from 20 normal male subjects ( all p < 0.0001) as shown in Figure 1. There was no difference in the 5 parameters of TGA between moderate and mild patients ( see Table 2 ). No correlations were noticed between HJHS score, Pettersson's score, HEAD-US and the lag phase, velocity index, peak time, peak thrombin and ETP among our cohort.

Conclusion(s): Our study demonstrates that there was no significant difference in the 5 parameters of TGA between moderate and mild patients and the 5 parameters of TGA in HA patients didn’t have good correlation with the severity of hemophilic arthropathy evaluated by three assessment tools.