Abstract
Background: Myelodysplastic syndromes (MDS) are a highly heterogenous group of diseases, which is refl ected in the need for a wide range of therapeutic approaches. At this time, the only curative option is allogeneic hematopoietic stem cell transplantation.
However, given the median age of MDS patients (around 70) and all the associated risks of transplantation, this remains a viable option for only a small percentage of patients. As such, the main therapeutic approaches are supportive therapy, growth factors, immunosuppression and, as of this century, the fi rst innovative approaches of immunomodulatory and hypomethylation therapy.
Yet, patient responses to most of these therapies (with the exception of immunomodulatory therapy for "5q- syndrome") do not exceed 40%. It is therefore imperative to continuously be looking for new promising approaches for MDS therapy.
Only in recent years, with advancements in the knowledge of pathogenesis and molecular genetics, new interesting drugs have begun to emerge. Purpose: This summarizing article provides an overview of new therapeutic options, even those in the early stages of clinical trials.