Therapeutic strategies in low and high-risk MDS: What does the future have to offer?

Abstract Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders characterized by cytopenias and increased risk of acute leukemia transformation. Prognosis of MDS patients can be assessed by various scoring systems, the most common being the International Prognostic Scoring System (IPSS) now refined by the revised version (IPSS-R). Genomic information at baseline, that is currently not included in clinical prognostic scores, will, in the future, help us to stratify patients with various prognoses. Therapy of MDS is based on risk stratification. The aim of therapy in low-risk MDS is to improve anemia or thrombocytopenia, decrease transfusion needs, improve quality of life, attempt to prolong overall survival, and reduce the risk of progression. In higher-risk MDS, the goal of therapy is to prolong survival and reduce the risk of transformation into acute leukemia. Only a few drugs are currently available for treatment, but more drugs are now under clinical investigation, in line with new, recently discovered molecular and immunological pathways. This review describes potential new drugs for low and high-risk MDS. The increasing knowledge of immunological and signalling pathways in MDS will assist us in identifying targeted patient-oriented treatments. In the near future, initial molecular stratification will lead the way to a personalized approach and targeted therapy.