Abstract Number: PB0543
Meeting: ISTH 2021 Congress
Background: Prophylaxis is the gold standard for the management of patients with severe haemophilia (PWSH) to avoid joint bleeds and other serious bleeding events. However, there is no international recommendation on optimal prophylactic regimen and dosing is still a crucial issue. We have previously reported that thrombin generation assay (TGA) better reflects the clinical bleeding tendency of PWSH and the test may be a better indicator of the efficacy of prophylaxis compared to FVIII:C.
Aims: The main objective was to compare trough TG capacity and FVIII levels in PWSH A and compare these two laboratory results with the number of spontaneous joint bleeds occurred during the last 6 months of prophylaxis.
Methods: This is an open label, multicentre, prospective cohort study including 34 PWSH A on prophylaxis with standard half-life FVIII concentrates. Trough TG and FVIII:C were measured and clinical data were collected.
Results: During the study period, nine patients (40% with hemophilic arthropathy) had spontaneous bleeds whilst on prophylaxis and 25 patients had no bleed. The mean trough FVIII:C of all patients was 2.18±2.9 IU/dL. Trough FVIII:C levels were not significantly different in the group who did not experienced spontaneous bleed (2.6±3.3 IU/dL) compared to patients who presented spontaneous bleeds (1±1.4 IU/dL ; p>0.05). We observed a tendency for lower ETP levels in the small group of 9 patients who had spontaneous bleeds while they were on prophylaxis (316±100 nM.min) compared to those who had no bleeding (500±379 nM.min). Fisher exact test showed that trough ETP level>516 nM.min had excellent negative predictive value for spontaneous bleeding risk (NPV=100% (95%CI=71.5–100); p=0.01) with a sensitivity 100% (95%CI=66–100) and specificity 44% (95%CI=24.4–65).
Conclusions: TGA may be valuable additional laboratory tool to FVIII:C, for the monitoring of prophylaxis in PWSH A and to personalize their treatment regimens.
To cite this abstract in AMA style:Meunier S, Lienhart A, Trossaert M, Gay V, Berger C, Tardy B, Volot F, Dargaud Y. Thrombin Generation Capacity of Patients with Severe Haemophilia Receiving Prophylaxis (HOPE Study) [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/thrombin-generation-capacity-of-patients-with-severe-haemophilia-receiving-prophylaxis-hope-study/. Accessed November 28, 2023.
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