Abstract

Myelodysplastic syndrome (MDS) is a group of clonal hematopoietic stem cell disorders and is uncommon in children comparing to adults. According to the proportion of blasts in the bone marrow (BM) and peripheral blood, it is divided into low-grade MDS and advanced MDS. Low-grade MDS often shows hypocellular BM, which makes differentiation from aplastic anemia and inherited bone marrow failure syndrome challenging. Treatment strategy for low-grade MDS should be determined based on the severity of cytopenias, karyotypes, and donor availability. In advanced MDS, two-thirds have chromosomal aberrations such as monosomy 7, and acute myeloid leukemia (AML) is the major differential diagnosis. Allogeneic hematopoietic cell transplantation (HCT) is the mainstay of treatment for children with advanced MDS; however, relapse and transplant-related mortality are obstacle to cure.

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