Myelodysplastic and myeloproliferative disorders in children

Myelodysplastic and myeloproliferative disorders are clonal myeloid malignancies characterized by the triad of a growth advantage of clonal cells, disturbed differentiation and increased apoptosis. The rarity of these disorders in children and the lack of a widely accepted classification have contributed to the paucity of reports on these malignancies in the pediatric literature. A number of significant advances have been achieved in recent years. The present review will focus on diagnostics and therapy.

International consensus has been achieved on classifying these disorders into three main groups; myelodysplastic syndrome (MDS), myeloid leukemia of Down syndrome (ML-DS) and juvenile myelomonocytic leukemia (JMML). In the last few years we have witnessed important advances, especially regarding the therapy of these disorders, and we have gained insights into the molecular pathogenesis of ML-DS and JMML.

Classification of myelodysplastic and myeloproliferative disorders has been facilitated. Chemotherapy regimens for ML-DS have been reduced, resulting in fewer toxic deaths and improved survival. The results of stem-cell transplantation for MDS and JMML have improved. Insight into the molecular mechanisms involved may open new therapeutic avenues.