The Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) are a heterogeneous group of malignant clonal hematopoietic stem cell disorders. MDS is characterized by ineffective hematopoiesis, marrow dysplasia, peripheral blood cytopenia, and a high propensity for transformation into AML. Although several agents that induce partial remissions and prolong survival in MDS patients have been identified, the only potential curative therapy for MDS is an allogeneic hematopoietic stem cell transplant (HSCT) for which most patients are ineligible due to advanced age, progressive disease, or comorbidities. There is also a small but increasingly recognized group of secondary MDS/AML which arises from inherited genetic diseases and evolves into an aggressive leukemia, often in young adulthood (such as those arising from germline mutations in GATA2 or RUNX1) for which pathogenesis and natural histories are poorly understood. A consequence of genotoxic therapy for a primary malignancy is therapy-related MDS/AML, which develops in a fraction of individuals who were cured of their original cancer, harbor high-risk mutations and typically are not sensitive to further chemotherapy.
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