Myelodysplastic syndromes in children: where are we today?

Myelodysplastic syndromes (MDSs) and other myeloproliferative disorders (MPDs) are rare entities, particularly in children. The latest classification of MPDs separates them into 3 major groups: MDS, juvenile myelomonocytic leukemia, and Down syndrome–associated myeloid leukemia. Although the blast count plays a central role in differentiating leukemias from MDSs, it is by no means sacrosanct; biological features as well as disease characteristics are also important considerations for diagnosis. Genetic alterations in MDSs have increasingly been noted in a majority of patients and when detected make diagnosis easy. Hematopoietic stem cell transplant is the treatment of choice, while newer agents have shown promise in adults and are presently in the advanced stages of clinical trials.