Real-world data of extended half-life factor VIII prophylaxis in patients with hemophilia A: A multicenter retrospective cohort study.

BACKGROUND

Extended half-life factor VIII (EHL-FVIII) products were developed to reduce infusion frequency and improve bleeding control in hemophilia A, but real-world data on switching from standard half-life FVIII (SHL-FVIII) prophylaxis remain limited. This study aimed to evaluate the effectiveness and treatment characteristics of EHL-FVIII prophylaxis in patients with hemophilia A in a real-world multicenter cohort.

METHODS

This multicenter retrospective cohort study included adult patients with hemophilia A from 16 hospitals in Türkiye who had received at least one year of SHL-FVIII prophylaxis followed by at least one year of EHL-FVIII prophylaxis. Each patient served as his own historical control. Bleeding outcomes during EHL-FVIII prophylaxis were compared with those recorded during the final 12 months of preceding SHL-FVIII prophylaxis. The primary outcome was the change in annualized bleeding rate (ABR), with annualized joint bleeding rate (AJBR) evaluated as an additional clinical outcome.

RESULTS

A total of 192 patients were included, with a median age of 28 years; 83.9% had severe hemophilia A. Following the switch from SHL-FVIII to EHL-FVIII prophylaxis, median ABR decreased from 3 (range, 0-22) to 1 (range, 0-14) (p < 0.001), while median AJBR decreased from 2 (range, 0-22) to 1 (range, 0-14) (p < 0.001). During EHL-FVIII prophylaxis, the median infusion frequency was 2 infusions per week, and 62% of patients received twice-weekly prophylaxis.

CONCLUSIONS

In this real-world multicenter cohort, switching from SHL-FVIII to EHL-FVIII prophylaxis improved bleeding outcomes in hemophilia A. EHL-FVIII products reduced infusion burden while maintaining effective bleed prevention, supporting their role in hemophilia management.