Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders characterized by ineffective clonal hematopoiesis responsible for cytopenia and an increased risk of transformation into acute myeloid leukemia. Although they mainly affect people over 60, their prognosis depends essentially on their cytogenetic and molecular characteristics. Two-thirds of patients are diagnosed with low-risk disease. In these patients, the aim of treatment is to improve cytopenias. New treatments are now available to improve cytopenias, particularly anemia, and several promising trials targeting the clone or inflammation are underway.
In high-risk patients, although hematopoietic stem cell transplantation is still the only curative option, hypomethylating agents remain the standard of treatment, but are unfortunately not curative. We are still waiting for the first therapeutic breakthrough that will change the outcome of these patients. Three Phase 3 trials, currently in the recruitment phase, offer such hope, combining a hypomethylating agent with venetoclax, sabatolimab or tamibarotene.
Keywords
Myelodysplastic syndromes, myelodysplastic neoplasms, low-risk MDS, high-risk MDS, hypomethylating agents, venetoclax, sabatolimab, tamibarotene