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治疗甲型血友病有多种凝血因子VIII产品可供选择:产品过多是好事还是坏事?

Many factor VIII products available in the treatment of hemophilia A: an embarrassment of riches?

作者信息

Lieuw Kenneth

机构信息

Department of Pediatrics, Walter Reed National Military Medical Center.

Department of Pediatrics, Uniformed Services University of the Health Sciences, Bethesda, MD, USA.

出版信息

J Blood Med. 2017 Jun 15;8:67-73. doi: 10.2147/JBM.S103796. eCollection 2017.

Abstract

Hemophilia A (HA) is a common bleeding disorder caused by the deficiency of factor VIII (FVIII) with an incidence of ~1 in 5000 male births. Replacement of FVIII is necessary to prevent and treat bleeding episodes. However, with multiple new drugs in addition to old standards, choosing among the different FVIII treatment options is harder than ever. There are FVIII products that are plasma derived or recombinant, FVIII products designed to extend the half-life of FVIII, and the first single-chain FVIII product, recombinant factor VIII single chain (rFVIII-SC). As development of inhibitors to FVIII continues to be a major problem in the care of HA patients, recent studies showing lower rates of inhibitor development with plasma-derived FVIIII products versus recombinant FVIII products have made choosing among the many options now available even more complex. Although still unproven, extended half-life (EHL) products may provide the hope of decreased immunogenicity but need further testing in previously untreated patients (PUPs). This review highlights some of the differences between FVIII products currently available and hopefully assists the clinician to decide which FVIII product to choose for their patients.

摘要

甲型血友病(HA)是一种常见的出血性疾病,由凝血因子VIII(FVIII)缺乏引起,在男性新生儿中的发病率约为1/5000。补充FVIII对于预防和治疗出血发作是必要的。然而,除了旧的标准疗法外,还有多种新药,在不同的FVIII治疗方案中进行选择比以往任何时候都更加困难。有血浆来源的或重组的FVIII产品、旨在延长FVIII半衰期的FVIII产品,以及首个单链FVIII产品,即重组因子VIII单链(rFVIII-SC)。由于FVIII抑制剂的产生仍然是HA患者治疗中的一个主要问题,最近的研究表明,与重组FVIII产品相比,血浆来源的FVIII产品产生抑制剂的比率较低,这使得在众多现有选择中进行抉择变得更加复杂。尽管仍未得到证实,但延长半衰期(EHL)产品可能有望降低免疫原性,但需要在既往未治疗的患者(PUPs)中进一步测试。本综述强调了目前可用的FVIII产品之间的一些差异,希望能帮助临床医生决定为其患者选择哪种FVIII产品。

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