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Prophylaxis for Hemophilia in the Era of Extended Half-Life Factor VIII/Factor IX Products

Berntorp, Erik LU and Andersson, Nadine G. (2016) In Seminars in Thrombosis and Hemostasis 42(5). p.518-525
Abstract

There are two main bioengineering approaches to extending the half-life of factor (F)VIII or FIX products used for hemophilia replacement therapy. These are fusion to Fc-immunoglobulin G (FVIII and FIX) or to albumin (FIX) or pegylation/glycopegylation (FVIII and FIX). Four FVIII and three FIX products are in clinical development or have recently been licensed in regions of the world. The reported half-life extension is approximately 1.5-fold for FVIII and 2.5-fold, or even longer, for FIX. Clinical trials have shown promising results with respect to extension of dose intervals and efficacy in the treatment and prevention of bleeding events. The role of these products in clinical practice has been discussed in terms of either improving... (More)

There are two main bioengineering approaches to extending the half-life of factor (F)VIII or FIX products used for hemophilia replacement therapy. These are fusion to Fc-immunoglobulin G (FVIII and FIX) or to albumin (FIX) or pegylation/glycopegylation (FVIII and FIX). Four FVIII and three FIX products are in clinical development or have recently been licensed in regions of the world. The reported half-life extension is approximately 1.5-fold for FVIII and 2.5-fold, or even longer, for FIX. Clinical trials have shown promising results with respect to extension of dose intervals and efficacy in the treatment and prevention of bleeding events. The role of these products in clinical practice has been discussed in terms of either improving convenience and adherence through prolongation of the interval between infusions or maintaining current intervals thereby increasing trough levels and the safety margin against bleeds. This review of extended half-life products addresses the possibilities and problems of their introduction in hemophilia treatment.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
factor IX, factor VIII, hemophilia A, hemophilia B, prophylaxis
in
Seminars in Thrombosis and Hemostasis
volume
42
issue
5
pages
8 pages
publisher
Thieme
external identifiers
  • Scopus:84976870484
ISSN
0094-6176
DOI
10.1055/s-0036-1571315
language
English
LU publication?
yes
id
7130ab66-78c7-421c-8094-9d265b8f1e84
date added to LUP
2016-07-18 08:46:23
date last changed
2016-10-13 05:11:44
@misc{7130ab66-78c7-421c-8094-9d265b8f1e84,
  abstract     = {<p>There are two main bioengineering approaches to extending the half-life of factor (F)VIII or FIX products used for hemophilia replacement therapy. These are fusion to Fc-immunoglobulin G (FVIII and FIX) or to albumin (FIX) or pegylation/glycopegylation (FVIII and FIX). Four FVIII and three FIX products are in clinical development or have recently been licensed in regions of the world. The reported half-life extension is approximately 1.5-fold for FVIII and 2.5-fold, or even longer, for FIX. Clinical trials have shown promising results with respect to extension of dose intervals and efficacy in the treatment and prevention of bleeding events. The role of these products in clinical practice has been discussed in terms of either improving convenience and adherence through prolongation of the interval between infusions or maintaining current intervals thereby increasing trough levels and the safety margin against bleeds. This review of extended half-life products addresses the possibilities and problems of their introduction in hemophilia treatment.</p>},
  author       = {Berntorp, Erik and Andersson, Nadine G.},
  issn         = {0094-6176},
  keyword      = {factor IX,factor VIII,hemophilia A,hemophilia B,prophylaxis},
  language     = {eng},
  month        = {07},
  number       = {5},
  pages        = {518--525},
  publisher    = {ARRAY(0xaf17e40)},
  series       = {Seminars in Thrombosis and Hemostasis},
  title        = {Prophylaxis for Hemophilia in the Era of Extended Half-Life Factor VIII/Factor IX Products},
  url          = {http://dx.doi.org/10.1055/s-0036-1571315},
  volume       = {42},
  year         = {2016},
}