Thrombin Generation in Pediatric Haemophilia A Patients on Extended Half‐Life FVIII versus Non‐FVIII Therapies

Introduction The therapeutic landscape for patients with haemophilia A (PwHA) is rapidly evolving with the introduction of extended half‐life FVIII (EHL‐FVIII) and non‐FVIII therapies that mimic FVIII, such as emicizumab (EMI). Monitoring non‐factor therapies in the laboratory poses challenges; however, the thrombin generation assay (TGA) can be utilized to evaluate hemostatic capacity. Aim To compare the endogenous thrombin potential (ETP) and peak thrombin (PT) in pediatric patients with moderate to severe haemophilia A (SHA) undergoing EHL‐FVIII therapies and EMI. Methods Platelet‐poor plasma (PPP) from PwHA on EHL‐FVIII or EMI prophylaxis was tested on the calibrated automated thrombogram (CAT) using PPP low reagent. Results ETP and PT were significantly higher in the EHL‐FVIII group compared to the EMI group. Conclusion Pediatric patients on EHL‐FVIII prophylaxis demonstrated higher ETP in vitro using PPP compared to those on EMI prophylaxis. These findings highlight the need for further systematic investigations to explore the implications of these differences in bleed control.