The myelodysplastic syndromes: The era of understanding

Abstract

Myelodysplastic syndromes are a heterogeneous group of clonal haematological stem cell disorders. Allogeneic stem cells transplantation remains the only curative treatment but only a minority of patients are eligible for this treatment. In spite of this, it has become clear that treatment with lenalidomide and azanucleotides can lead to increased overall survival in particular subsets of patients with MDS. The relative silence on the therapeutic side is counter-balanced by major advances in the understanding of this heterogeneous disease. The introduction of high-throughput molecular techniques has resulted in the discovery that most patients harbour molecular aberrations, including pathways such as the spliceosome machinery previously not known to be involved. These newly discovered pathways will undoubtedly result in new therapeutic strategies for this difficult to treat disease.