Clinical and Surgical Experiences with Efmoroctocog Alfa in Pediatric Population with Hemophilia A
Clinical and Applied Thrombosis/Hemostasis, cilt.31, 2025 (SCI-Expanded, Scopus)
- Yayın Türü: Makale / Tam Makale
- Cilt numarası: 31
- Basım Tarihi: 2025
- Doi Numarası: 10.1177/10760296251377646
- Dergi Adı: Clinical and Applied Thrombosis/Hemostasis
- Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, EMBASE, MEDLINE, Directory of Open Access Journals
- Anahtar Kelimeler: hemophilia A, bleeding, surgery, extended half life, efmoroktocog alfa
- Atatürk Üniversitesi Adresli: Evet
Özet
Introduction: Hemophilia A is a rare inherited bleeding disorder. Less than 1% of FVIII level in plasma is classified as severe, 1%–5% as moderate, and above 5% as mild disease. The aim of this study is to share clinical and surgical experiences in Hemophilia A with extended half-life (EHL) recombinant FVIII preparations. Material and Methods: A retrospective observational study between 2024 and 2025, inpatient or outpatient follow-up was carried out in the Pediatric Hematology clinic of our university hospital. A total of 18 patients diagnosed with severe FVIII deficiency were included in this study. Results: The average age of the patients was 8.4 years (min. 20 months—max. 17 years). All patients were male and inhibitor negative severe FVIII deficiecy patients. Patients were numbered as P1-18 respectively. P1, P4, and P8 are 20 months, 3 years, and 4 years old respectively. They were evaluated as previously untreated patients (PUP) with a new diagnosis of severe FVIII deficiency and the other patients were previously treated patients (PTPs). P10 had 2 infected decayed molars and 1 incisor extracted in the same dental session. P13 underwent circumcision operation. P18 had refixation surgery due to a left arm radius and ulna bone fracture, and a cast was applied. All three surgical operations were performed under treatment of recombinant FVIII Efmoroctocog alfa (Ealfa/Elocta®) successfully. Conclusion: Efmoroctocog alfa seems to be effective in EHL FVIII replacement therapy and surgical management of hemophilia A. Compared with standard half-life (SHL) FVIII products, EHL FVIII products such as Ealfa have the potential to optimize prophylactic outcomes by decreasing the burden of treatment or increasing the level of bleed protection.