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.

• Keywords
• Diamond-Blackfan anaemia, GATA1, autophagy, c-myc, erythropoiesis, glucocorticosteroid, mTOR,
• MeSH
• Anemia, Diamond-Blackfan drug therapy   metabolism   MeSH
• Erythropoiesis drug effects   MeSH
• Gene Regulatory Networks drug effects   MeSH
• Glucocorticoids pharmacology   therapeutic use   MeSH
• Humans MeSH
• Gene Expression Regulation drug effects   MeSH
• Treatment Outcome MeSH
• Check Tag
• Humans MeSH
• Publication type
• Journal Article MeSH
• Review MeSH
• Names of Substances
• Glucocorticoids MeSH