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Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia.

Sjögren, Sara LU and Flygare, Johan LU (2012) In Scientific World Journal 2012.
Abstract
Diamond-Blackfan anemia (DBA) is a congenital erythroid hypoplastic anemia, characterized by macrocytic anemia, reticulocytopenia, and severely reduced numbers of erythroid precursors in the bone marrow. For more than fifty years, glucocorticoids have remained the main option for pharmacological treatment of DBA. While continuous glucocorticoid administration increases hemoglobin levels in a majority of DBA patients, it also causes severe side effects. There is therefore a great need for more specific and effective treatments to boost or replace the use of glucocorticoids. Over the years, many alternative therapies have been tried out, but most of them have shown to be ineffective. Here we review previous and current attempts to develop... (More)
Diamond-Blackfan anemia (DBA) is a congenital erythroid hypoplastic anemia, characterized by macrocytic anemia, reticulocytopenia, and severely reduced numbers of erythroid precursors in the bone marrow. For more than fifty years, glucocorticoids have remained the main option for pharmacological treatment of DBA. While continuous glucocorticoid administration increases hemoglobin levels in a majority of DBA patients, it also causes severe side effects. There is therefore a great need for more specific and effective treatments to boost or replace the use of glucocorticoids. Over the years, many alternative therapies have been tried out, but most of them have shown to be ineffective. Here we review previous and current attempts to develop such alternative therapies for DBA. We further discuss how emerging knowledge regarding the pathological mechanism in DBA and the therapeutic mechanism of glucocorticoids treatment may reveal novel drug targets for DBA treatment. (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
Scientific World Journal
volume
2012
publisher
Hindawi Publishing Corporation
external identifiers
  • WOS:000304266200001
  • PMID:22619618
  • Scopus:84861064000
ISSN
2356-6140
DOI
10.1100/2012/184362
language
English
LU publication?
yes
id
d99b04f7-9eaa-4b91-86c7-c0cf08c74e65 (old id 2608584)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/22619618?dopt=Abstract
date added to LUP
2012-06-03 20:56:56
date last changed
2017-01-08 05:33:47
@article{d99b04f7-9eaa-4b91-86c7-c0cf08c74e65,
  abstract     = {Diamond-Blackfan anemia (DBA) is a congenital erythroid hypoplastic anemia, characterized by macrocytic anemia, reticulocytopenia, and severely reduced numbers of erythroid precursors in the bone marrow. For more than fifty years, glucocorticoids have remained the main option for pharmacological treatment of DBA. While continuous glucocorticoid administration increases hemoglobin levels in a majority of DBA patients, it also causes severe side effects. There is therefore a great need for more specific and effective treatments to boost or replace the use of glucocorticoids. Over the years, many alternative therapies have been tried out, but most of them have shown to be ineffective. Here we review previous and current attempts to develop such alternative therapies for DBA. We further discuss how emerging knowledge regarding the pathological mechanism in DBA and the therapeutic mechanism of glucocorticoids treatment may reveal novel drug targets for DBA treatment.},
  articleno    = {184362},
  author       = {Sjögren, Sara and Flygare, Johan},
  issn         = {2356-6140},
  language     = {eng},
  publisher    = {Hindawi Publishing Corporation},
  series       = {Scientific World Journal},
  title        = {Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia.},
  url          = {http://dx.doi.org/10.1100/2012/184362},
  volume       = {2012},
  year         = {2012},
}