VIII 因子替代疗法仍然是 A 型血友病的标准治疗方法。
Factor VIII replacement is still the standard of care in haemophilia A.
机构信息
Division of Hematology/Oncology, Icahn School of Medicine, Mount Sinai, New York, NY, United States of America.
Scientific Direction, IRCCS Foundation Ca' Granda Maggiore Policlinico Hospital, Milan, Italy.
出版信息
Blood Transfus. 2019 Nov;17(6):479-486. doi: 10.2450/2019.0211-19. Epub 2019 Dec 11.
Abstract
Prophylactic factor VIII (FVIII) has dramatically improved haemophilia A treatment, preventing joint bleeding and halting the deterioration of joint status. FVIII products with an extended plasma half-life further improve patients' quality of life and increase therapeutic adherence. New licensed classes of non-replacement products include prophylactic emicizumab, which is administered subcutaneously up to every 4 weeks. However, this drug is not suitable for acute bleeding episodes or management of major surgery, and long-term data on the impact of emicizumab on joint health, FVIII inhibitor development and thrombotic risk are awaited. Prophylaxis with FVIII replacement remains the standard of care in haemophilia A, with the aim of achieving a level of haemostasis control that allows patients to meet their lifestyle goals.
摘要
预防性因子 VIII（FVIII）的使用显著改善了 A 型血友病的治疗效果，预防了关节出血，并阻止了关节状况的恶化。具有延长血浆半衰期的 FVIII 产品进一步提高了患者的生活质量并增加了治疗的依从性。新型非替代产品的许可类别包括预防性依库珠单抗，该药可每 4 周皮下给药一次。然而，该药并不适用于急性出血发作或大型手术的处理，且关于依库珠单抗对关节健康、FVIII 抑制剂发展和血栓形成风险影响的长期数据仍在等待中。FVIII 替代物的预防性治疗仍然是 A 型血友病的护理标准，目的是达到止血控制水平，使患者能够实现其生活方式目标。
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