During the last decade, substantial advances have been made in the understanding of
the complex molecular, immunological and cellular disturbances involved in the initiation as well
as evolution of myelodysplastic syndromes (MDS). In 85% of the mainly frail and older patient
population, anemia is present at the time of diagnosis and is thus a major therapeutic challenge.
High rates of primary resistance to erythropoiesis-stimulating agents (ESAs), the currently only
approved standard therapy to treat anemia in lower-risk MDS, demand the development of novel
and effcient drugs with a good safety profile. Luspatercept, a ligand trap of activin receptor II, is able
to promote late stage erythropoiesis even in patients failing prior ESA treatment. The presence of
ring sideroblastic phenotype defines a subgroup of patients with higher response rates. Additionally,
recent developments in clinical research using HIF-1 or telomerase modulation by roxadustat or
imetelstat are promising. Other areas of translational research involve targeting the inflammasome
by anti-inflammatory drugs in order to improve anemia. These efforts will hopefully pave the way
for new targeted treatment options for anemic low-risk MDS patients.
| Identifer | oai:union.ndltd.org:DRESDEN/oai:qucosa:de:qucosa:89036 |
| Date | 16 January 2024 |
| Creators | Kubasch, Anne Sophie, Platzbecker, Uwe |
| Publisher | MDPI |
| Source Sets | Hochschulschriftenserver (HSSS) der SLUB Dresden |
| Language | English |
| Detected Language | English |
| Type | info:eu-repo/semantics/publishedVersion, doc-type:article, info:eu-repo/semantics/article, doc-type:Text |
| Rights | info:eu-repo/semantics/openAccess |
| Relation | 3853 |
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