a NHLS and the Charlotte Maxeke , Johannesburg academic Hospital, University of the Witwatersrand , Johannesburg , South Africa.
Expert Rev Hematol. 2018 Dec;11(12):937-943. doi: 10.1080/17474086.2018.1549478. Epub 2018 Dec 3.
: rFVIIIFC was the first extended half-life product to complete the phase 3 development program and be registered. It was developed to reduce the high treatment burden imposed by prophylaxis. It is now one of four extended half-life products available for a variety of indications in hemophilia A. This article focus on the efficacy use of rFVIIIFC in the prevention of bleeds in hemophilia A. : This article provides an update on efficacy data from three clinical studies describing the use of rFVIIIFC in the treatment and prevention of bleeds in hemophilia A. The update includes the efficacy use of rFVIII in all age groups, in the perisurgical setting, in immune tolerance induction, and in improving the quality of life of patients. The role of rFVIIIFC prophylaxis in the face of rapidly evolving non-replacement therapy and gene therapy is summarized. : The role of rFVIIIFC in hemophilia A prophylaxis is uncertain in the light of development of newer prophylaxis agents with better route of administration, improved pharmacokinetic and superior efficacy profiles. While rFVIIIFC was primarily developed for prophylaxis in hemophilia A, this role may change in the face of competitive extended half-life products and non-replacement therapies.
rFVIIIFC 是首个完成 3 期开发项目并注册的延长半衰期产品。它的开发旨在降低预防治疗带来的高治疗负担。它现在是四种可用于多种 A 型血友病适应证的延长半衰期产品之一。本文重点介绍 rFVIIIFC 在预防 A 型血友病出血方面的疗效。
本文提供了三项临床研究的疗效数据更新，这些研究描述了 rFVIIIFC 在 A 型血友病出血的治疗和预防中的应用。更新内容包括 rFVIII 在所有年龄组、围手术期、免疫耐受诱导以及改善患者生活质量方面的疗效。总结了 rFVIIIFC 在面对快速发展的非替代治疗和基因治疗时的预防作用。
鉴于具有更好给药途径、改善药代动力学和更优疗效特征的新型预防药物的发展，rFVIIIFC 在 A 型血友病预防中的作用尚不确定。虽然 rFVIIIFC 主要是为 A 型血友病的预防而开发的，但在面对竞争的延长半衰期产品和非替代治疗时，这种作用可能会发生变化。
