Myelodysplastic syndromes (MDS) are a collection of hematopoietic stem cell (HSC) disorders that consist of blood cytopenias, marrow dysplasia, and a predisposition to acute myeloid leukemia (AML). Approximately 30% of MDS patients go on to develop aggressive AML. MDS is fatal in a majority of patients as a result of marrow failure, immune dysfunction, and/or transformation to overt leukemia. Allogeneic HSC transplantation can be curative in MDS, but this option is suitable only in the small proportion of younger patients. Alterative treatment options for MDS include demethylating agents and immunomodulatory therapies. Disappointingly, the efficacy and durability of the remaining treatment options is variable. Targeted therapies have been effective in multiple myeloid diseases, and may also be effective in
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Beverly et al. (2014) studied this question.
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