Myelodysplastic syndromes: Contemporary review and how we treat

American Journal of Hematology
Naseema GangatAyalew Tefferi

Abstract

Myelodysplastic syndromes (MDS) are a heterogeneous group of clonal stem cell disorders with an inherent tendency for leukemic transformation. Diagnosis is currently based on the presence of peripheral blood cytopenias, peripheral blood and bone marrow dysplasia/blasts, and clonal cytogenetic abnormalities. With the advent of next generation sequencing, recurrent somatic mutations in genes involved in epigenetic regulation (TET2, ASXL1, EZH2, DNMT3A, IDH1/2), RNA splicing (SF3B1, SRSF2, U2AF1, ZRSR2), DNA damage response (TP53), transcriptional regulation (RUNX1, BCOR, ETV6) and signal transduction (CBL, NRAS, JAK2) have been identified in MDS. Conventional prognostication is by the revised International prognostic scoring system (IPSS-R) with additional adverse prognosis conferred by presence of ASXL1, EZH2, or TP53 mutations. Currently Food and Drug administration (FDA)-approved drugs for the treatment of MDS are not curative and their effect on survival is limited; they include the hypomethylating agents (HMA) azacitidine and decitabine and lenalidomide for MDS with isolated del(5q). To date, allogeneic stem cell transplant (ASCT) remains the only treatment option for possible cure. Given the current lack of drugs with convi...Continue Reading

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Citations

Apr 22, 2016·Expert Review of Hematology·Morena CairaCorrado Girmenia
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Aug 29, 2018·American Journal of Hematology·Dame IdossaAyalew Tefferi
Jul 25, 2018·American Journal of Hematology·Ayalew Tefferi
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Jan 15, 2019·American Journal of Hematology·Ayalew TefferiNaseema Gangat
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