Advanced

New treatments in hemophilia: insights for the clinician.

Knobe, Karin LU and Berntorp, Erik LU (2012) In Therapeutic advances in hematology 3(3). p.165-175
Abstract
Hemophilia has evolved from an often fatal hereditary bleeding disorder to a disorder for which safe and effective treatment is available. However, there are several challenges remaining in the treatment of hemophilia. Prophylaxis to prevent bleeding is costly and requires frequent intravenous injections, which are cumbersome for patients. Venous access is often difficult to achieve, especially in small children where central venous lines may need to be implanted. Development of inhibitory antibodies makes treatment of acute bleeds difficult and prophylaxis in patients with inhibitors must also be better addressed. In order to improve treatment, new products are being developed, some of which are already in clinical trials. There are... (More)
Hemophilia has evolved from an often fatal hereditary bleeding disorder to a disorder for which safe and effective treatment is available. However, there are several challenges remaining in the treatment of hemophilia. Prophylaxis to prevent bleeding is costly and requires frequent intravenous injections, which are cumbersome for patients. Venous access is often difficult to achieve, especially in small children where central venous lines may need to be implanted. Development of inhibitory antibodies makes treatment of acute bleeds difficult and prophylaxis in patients with inhibitors must also be better addressed. In order to improve treatment, new products are being developed, some of which are already in clinical trials. There are several approaches to prolonging half-lives such as PEGylation, Fc fusion and albumin fusion. Increased activity has been demonstrated in preclinical trials for factor IX and in a human trial with factor VII where the activity of the molecules has been increased by manipulation of the molecular composition. Additional approaches, including blockage of inhibitors of clotting, are also under investigation. Factor VIII and factor IX gene therapy have become a tangible possibility since phase I data recently have been published. Results are promising and there is hope that in the near future substantial progress will be made, perhaps making hemophilia the first genetic condition to be cured. (Less)
Please use this url to cite or link to this publication:
author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Therapeutic advances in hematology
volume
3
issue
3
pages
165 - 175
external identifiers
  • PMID:23556123
ISSN
2040-6207
DOI
10.1177/2040620712440007
language
English
LU publication?
yes
id
246f1c3c-a757-48b5-b16e-7e8d98bbb924 (old id 3734163)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/23556123?dopt=Abstract
date added to LUP
2013-05-02 12:36:43
date last changed
2016-09-20 03:31:41
@misc{246f1c3c-a757-48b5-b16e-7e8d98bbb924,
  abstract     = {Hemophilia has evolved from an often fatal hereditary bleeding disorder to a disorder for which safe and effective treatment is available. However, there are several challenges remaining in the treatment of hemophilia. Prophylaxis to prevent bleeding is costly and requires frequent intravenous injections, which are cumbersome for patients. Venous access is often difficult to achieve, especially in small children where central venous lines may need to be implanted. Development of inhibitory antibodies makes treatment of acute bleeds difficult and prophylaxis in patients with inhibitors must also be better addressed. In order to improve treatment, new products are being developed, some of which are already in clinical trials. There are several approaches to prolonging half-lives such as PEGylation, Fc fusion and albumin fusion. Increased activity has been demonstrated in preclinical trials for factor IX and in a human trial with factor VII where the activity of the molecules has been increased by manipulation of the molecular composition. Additional approaches, including blockage of inhibitors of clotting, are also under investigation. Factor VIII and factor IX gene therapy have become a tangible possibility since phase I data recently have been published. Results are promising and there is hope that in the near future substantial progress will be made, perhaps making hemophilia the first genetic condition to be cured.},
  author       = {Knobe, Karin and Berntorp, Erik},
  issn         = {2040-6207},
  language     = {eng},
  number       = {3},
  pages        = {165--175},
  series       = {Therapeutic advances in hematology},
  title        = {New treatments in hemophilia: insights for the clinician.},
  url          = {http://dx.doi.org/10.1177/2040620712440007},
  volume       = {3},
  year         = {2012},
}