Solgun Hüseyin Avni, Ozay Mustafa
SBU Kanuni Sultan Suleyman Training and Research Hospital, Pediatric Hematology and Oncology, Istanbul, Turkiye.
SBU Gaziantep City Hospital, Pediatric Hematolgoy and Oncology, Gaziantep, Turkiye.
Clin Appl Thromb Hemost. 2025 Jan-Dec;31:10760296251377646. doi: 10.1177/10760296251377646. Epub 2025 Sep 22.
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.
引言
甲型血友病是一种罕见的遗传性出血性疾病。血浆中FVIII水平低于1%被归类为重度，1%-5%为中度，高于5%为轻度疾病。本研究的目的是分享使用延长半衰期（EHL）重组FVIII制剂治疗甲型血友病的临床和手术经验。
材料和方法
2024年至2025年进行了一项回顾性观察研究，在我们大学医院的儿科血液科进行住院或门诊随访。本研究共纳入18例诊断为重度FVIII缺乏的患者。
结果
患者的平均年龄为8.4岁（最小20个月 - 最大17岁）。所有患者均为男性且无抑制物的重度FVIII缺乏患者。患者分别编号为P1 - 18。P1、P4和P8分别为20个月、3岁和4岁。他们被评估为新诊断为重度FVIII缺乏的既往未治疗患者（PUP），其他患者为既往治疗患者（PTP）。P10在同一次牙科治疗中拔除了2颗感染的乳磨牙和1颗门牙。P13接受了包皮环切手术。P18因左臂桡骨和尺骨骨折进行了内固定手术，并应用了石膏。所有这三台手术均在重组FVIII依洛凝血素α（Ealfa/Elocta）治疗下成功完成。
结论
依洛凝血素α似乎在EHL FVIII替代治疗和甲型血友病的手术管理中有效。与标准半衰期（SHL）FVIII产品相比，Ealfa等EHL FVIII产品有可能通过减轻治疗负担或提高出血保护水平来优化预防效果。
