Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems

Diamond-Blackfan anaemia (DBA) is a red blood cell aplasia that in the majority of cases is associated with ribosomal protein (RP) aberrations. However, the mechanism by which this disorder leads to such a specific phenotype remains unclear. Even more elusive is the reason why non-specific agents su...

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Autores principales: Macečková, Zuzana, Kubíčková, Agáta, De Sanctis, Juan Bautista, Hajdúch, Marian
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8837118/
https://www.ncbi.nlm.nih.gov/pubmed/35163808
http://dx.doi.org/10.3390/ijms23031886
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author Macečková, Zuzana
Kubíčková, Agáta
De Sanctis, Juan Bautista
Hajdúch, Marian
author_facet Macečková, Zuzana
Kubíčková, Agáta
De Sanctis, Juan Bautista
Hajdúch, Marian
author_sort Macečková, Zuzana
collection PubMed
description Diamond-Blackfan anaemia (DBA) is a red blood cell aplasia that in the majority of cases is associated with ribosomal protein (RP) aberrations. However, the mechanism by which this disorder leads to such a specific phenotype remains unclear. Even more elusive is the reason why non-specific agents such as glucocorticosteroids (GCs), also known as glucocorticoids, are an effective therapy for DBA. In this review, we (1) explore why GCs are successful in DBA treatment, (2) discuss the effect of GCs on erythropoiesis, and (3) summarise the GC impact on crucial pathways deregulated in DBA. Furthermore, we show that GCs do not regulate DBA erythropoiesis via a single mechanism but more likely via several interdependent pathways.
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spelling pubmed-88371182022-02-12 Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems Macečková, Zuzana Kubíčková, Agáta De Sanctis, Juan Bautista Hajdúch, Marian Int J Mol Sci Review Diamond-Blackfan anaemia (DBA) is a red blood cell aplasia that in the majority of cases is associated with ribosomal protein (RP) aberrations. However, the mechanism by which this disorder leads to such a specific phenotype remains unclear. Even more elusive is the reason why non-specific agents such as glucocorticosteroids (GCs), also known as glucocorticoids, are an effective therapy for DBA. In this review, we (1) explore why GCs are successful in DBA treatment, (2) discuss the effect of GCs on erythropoiesis, and (3) summarise the GC impact on crucial pathways deregulated in DBA. Furthermore, we show that GCs do not regulate DBA erythropoiesis via a single mechanism but more likely via several interdependent pathways. MDPI 2022-02-08 /pmc/articles/PMC8837118/ /pubmed/35163808 http://dx.doi.org/10.3390/ijms23031886 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Macečková, Zuzana
Kubíčková, Agáta
De Sanctis, Juan Bautista
Hajdúch, Marian
Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems
title Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems
title_full Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems
title_fullStr Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems
title_full_unstemmed Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems
title_short Effect of Glucocorticosteroids in Diamond-Blackfan Anaemia: Maybe Not as Elusive as It Seems
title_sort effect of glucocorticosteroids in diamond-blackfan anaemia: maybe not as elusive as it seems
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8837118/
https://www.ncbi.nlm.nih.gov/pubmed/35163808
http://dx.doi.org/10.3390/ijms23031886
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