Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia.
(2012) In The 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:
https://lup.lub.lu.se/record/2608584
- author
- Sjögren, Sara LU and Flygare, Johan LU
- organization
- publishing date
- 2012
- type
- Contribution to journal
- publication status
- published
- subject
- in
- The Scientific World Journal
- volume
- 2012
- article number
- 184362
- publisher
- Hindawi Limited
- external identifiers
-
- wos:000304266200001
- pmid:22619618
- scopus:84861064000
- pmid:22619618
- 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
- 2016-04-04 09:33:18
- date last changed
- 2024-01-12 15:07:09
@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.}},
author = {{Sjögren, Sara and Flygare, Johan}},
issn = {{2356-6140}},
language = {{eng}},
publisher = {{Hindawi Limited}},
series = {{The Scientific World Journal}},
title = {{Progress towards Mechanism-Based Treatment for Diamond-Blackfan Anemia.}},
url = {{http://dx.doi.org/10.1100/2012/184362}},
doi = {{10.1100/2012/184362}},
volume = {{2012}},
year = {{2012}},
}