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Prevention and Management of Bleeding Episodes in Children with Hemophilia

Ljung, Rolf C.R. LU (2018) In Pediatric Drugs 20(5). p.455-464
Abstract

Regular prophylactic treatment with factor VIII (FVIII) and factor IX (FIX) concentrates in hemophilia A and B, respectively, is introduced in early infancy and has resulted in dramatic improvement of the conditions. Recombinant FVIII and FIX concentrates have been available for > 25 years and have been modified and refined through the years; however, unfortunately frequent intravenous administrations are still necessary. The half-lives of these products have now been extended (EHL) by fusion with albumin, the Fc-portion of IgG, or by being PEGylated. This has been very successful for EHL-FIX, with 3–5 times longer half-life, and to a lesser degree for EHL-FVIII with a half-life extension of only 1.5 times the conventional products.... (More)

Regular prophylactic treatment with factor VIII (FVIII) and factor IX (FIX) concentrates in hemophilia A and B, respectively, is introduced in early infancy and has resulted in dramatic improvement of the conditions. Recombinant FVIII and FIX concentrates have been available for > 25 years and have been modified and refined through the years; however, unfortunately frequent intravenous administrations are still necessary. The half-lives of these products have now been extended (EHL) by fusion with albumin, the Fc-portion of IgG, or by being PEGylated. This has been very successful for EHL-FIX, with 3–5 times longer half-life, and to a lesser degree for EHL-FVIII with a half-life extension of only 1.5 times the conventional products. New treatment principles using FVIII mimetics or monoclonal antibodies that rebalance the pro- and anti-coagulation system by interfering with production of anti-thrombin or tissue factor pathway inhibitor have the benefits of long-lasting activity, subcutaneous administration, and being useful in patients both with and without neutralizing antibodies. As the ultimate treatment, recent progress has also been made with gene therapy of both hemophilia A and B.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Pediatric Drugs
volume
20
issue
5
pages
455 - 464
publisher
Adis International Ltd
external identifiers
  • scopus:85051834700
ISSN
1174-5878
DOI
10.1007/s40272-018-0307-z
language
English
LU publication?
yes
id
aea5feb2-2b26-47ae-ae8f-fa052b09c00f
date added to LUP
2018-09-12 09:43:55
date last changed
2019-08-14 04:22:27
@article{aea5feb2-2b26-47ae-ae8f-fa052b09c00f,
  abstract     = {<p>Regular prophylactic treatment with factor VIII (FVIII) and factor IX (FIX) concentrates in hemophilia A and B, respectively, is introduced in early infancy and has resulted in dramatic improvement of the conditions. Recombinant FVIII and FIX concentrates have been available for &gt; 25 years and have been modified and refined through the years; however, unfortunately frequent intravenous administrations are still necessary. The half-lives of these products have now been extended (EHL) by fusion with albumin, the Fc-portion of IgG, or by being PEGylated. This has been very successful for EHL-FIX, with 3–5 times longer half-life, and to a lesser degree for EHL-FVIII with a half-life extension of only 1.5 times the conventional products. New treatment principles using FVIII mimetics or monoclonal antibodies that rebalance the pro- and anti-coagulation system by interfering with production of anti-thrombin or tissue factor pathway inhibitor have the benefits of long-lasting activity, subcutaneous administration, and being useful in patients both with and without neutralizing antibodies. As the ultimate treatment, recent progress has also been made with gene therapy of both hemophilia A and B.</p>},
  author       = {Ljung, Rolf C.R.},
  issn         = {1174-5878},
  language     = {eng},
  month        = {08},
  number       = {5},
  pages        = {455--464},
  publisher    = {Adis International Ltd},
  series       = {Pediatric Drugs},
  title        = {Prevention and Management of Bleeding Episodes in Children with Hemophilia},
  url          = {http://dx.doi.org/10.1007/s40272-018-0307-z},
  volume       = {20},
  year         = {2018},
}