Precision medicine for MDS, what drugs for which patients?

David Steensma, MD of the Dana-Faber Cancer institute, Boston, MA gives an overview of his talk on precision medicine for myelodysplastic syndromes (MDS) held at the 2016 Annual Meeting of the British Society of Haematology (BSH) and International Society of Hematology (ISH) in Glasgow, Scotland. Dr Steensma discusses how precision medicine can be interpreted differently and more specifically, what drug therapies could be targeted to which patients with MDS. Dr Steensma discusses what is being learned about the genetics of MDS and how to refine treatment algorithms in the future. He further talks about the discovery that molecular diagnostic testing helps with diagnosis in ambiguous cases and with prognostic stratification, but has so far had little effect on choosing a particular drug for a particular patient. He discusses how patients with TET2 mutations respond marginally better to azacitidine or decitabine therapy than those that do not have such mutations but that the difference is not striking enough to make a clinical decision based upon it. Decisions can, however, be based on p53 mutations particularly in association with a complex karyotype as affected patients seem to do very poorly with reduced intensity conditioning stem cell transplantation. Dr Steensma suggests that new approaches to treatment are needed to improve the outcome for these patients. Finally, he speaks about what can be done to help improve outcomes in patients undergoing stem cell transplantation who have high risk. This includes immunotherapies such as the adaptation of checkpoint inhibitors pre- or post-transplant, specific targeted agents, which where available, could be resumed post-transplant to decrease the risk of relapse and the manipulation of immune cells to better target the tumor cells. CAR T-cells are the best examples of this, however, there may be other manipulations that could be done to improve the outcomes.

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