Profile of efraloctocog alfa and its potential in the treatment of hemophilia A

被引:13
作者
George, Lindsey A. [1 ,2 ]
Camire, Rodney M. [1 ,2 ,3 ]
机构
[1] Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA
[2] Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA
[3] Univ Penn, Perelman Sch Med, Dept Pediat, Philadelphia, PA 19104 USA
来源
JOURNAL OF BLOOD MEDICINE | 2015年 / 6卷
关键词
hemophilia A; factor VIII; Fc fusion; bioengineered products; efraloctocog alfa;
D O I
10.2147/JBM.S54632
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Hemophilia care has improved dramatically over the past 50 years, evolving from plasma concentrates, to purified plasma proteins, to recombinant clotting factors. These collective developments allowed for home delivery of on-demand and prophylactic treatment, resulting in the reduction of hemophilia morbidity and mortality and improved quality of life. Although efficacious in treating bleeding, conventional factor products' half-lives require frequent venipuncture, which remains a significant burden to patients. Despite the remarkable advances in hemophilia care, no improvements have, until now, been made to the pharmacokinetic properties of factor products. Multiple strategies have more recently been employed to generate novel bioengineered products that, with great hope, represent the next wave of progress in hemophilia care. The use of these products will undoubtedly raise important discussion about choosing conventional factor over new long-acting factor products. Incorporation of these therapies into clinical care is accompanied by unanswered safety questions that will likely be evaluated only in postmarketingsurveillance analysis. Further, these products may change current treatment paradigms with unclear cost repercussions and feasibility. This paper will review efraloctocog alfa (FVIII-Fc) and its role in the treatment of hemophilia A.
引用
收藏
页码:131 / 141
页数:11
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