Klin Onkol 2021; 34(5): 356-365. DOI: 10.48095/ccko2021356.
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.
