Real-world data of extended half-life factor VIII prophylaxis in patients with hemophilia A: A multicenter retrospective cohort study
Thrombosis Research, cilt.266, 2026 (SCI-Expanded, Scopus)
- Yayın Türü: Makale / Tam Makale
- Cilt numarası: 266
- Basım Tarihi: 2026
- Doi Numarası: 10.1016/j.thromres.2026.109871
- Dergi Adı: Thrombosis Research
- Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, BIOSIS, Chemical Abstracts Core, EMBASE, MEDLINE, Academic Search Ultimate (EBSCO)
- Anahtar Kelimeler: Efmoroctocog alfa, Extended half-life factor VIII, Hemophilia A, Rurioctocog alfa pegol, Turoctocog alfa pegol
- Van Yüzüncü Yıl Üniversitesi Adresli: Evet
Özet
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.