Clinical and Surgical Experiences with Efmoroctocog Alfa in Pediatric Population with Hemophilia A.
Аннотация
IntroductionHemophilia 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 MethodsA 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.ResultsThe 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.ConclusionEfmoroctocog 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.
Предметные рубрики MeSH
Подготовка и перевод публикации
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
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