Beschreibung:
During the last decade, substantial advances have been made in the understanding ofthe complex molecular, immunological and cellular disturbances involved in the initiation as wellas evolution of myelodysplastic syndromes (MDS). In 85% of the mainly frail and older patientpopulation, 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 onlyapproved standard therapy to treat anemia in lower-risk MDS, demand the development of noveland effcient drugs with a good safety profile. Luspatercept, a ligand trap of activin receptor II, is ableto promote late stage erythropoiesis even in patients failing prior ESA treatment. The presence ofring 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 orimetelstat are promising. Other areas of translational research involve targeting the inflammasomeby anti-inflammatory drugs in order to improve anemia. These efforts will hopefully pave the wayfor new targeted treatment options for anemic low-risk MDS patients.