Treatment of myelodysplastic syndromes in the era of precision medicine and immunomodulatory drugs: a focus on higher-risk disease

Abstract

Myelodysplastic syndromes (MDS) are a heterogeneous clonal disease of myeloid neoplasms characterized by ineffective hematopoiesis, variable degree of cytopenias, and an increased risk of progression to acute myeloid leukemia (AML). Molecular and genetic characterization of MDS has led to a better understanding of the disease pathophysiology and is leading to the development of novel therapies. Targeted and immune therapies have shown promising results in different hematologic malignancies. However, their potential use in MDS is yet to be fully defined. Here, we review the most recent advances in therapeutic approaches in MDS, focusing on higher-risk disease. Allogeneic hematopoietic cell transplantation is beyond the scope of this article. © 2022, The Author(s).

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Keywords

High-risk mds, Immune dysregulation, Immunotherapy, Myeloid malignancy, Precision medicine, Targeted therapy, Azacitidine, Cd135 antigen, Cedazuridine plus decitabine, Cytarabine plus daunorubicin, Decitabine, Durvalumab, Enasidenib, Eprenetapopt, Gilteritinib, Guadecitabine, Immunomodulating agent, Ipilimumab, Isocitrate dehydrogenase 1, Isocitrate dehydrogenase 2, Ivosidenib, Lenalidomide, Luspatercept, Magrolimab, Nivolumab, Olutasidenib, Pembrolizumab, Pevonedistat, Sabatolimab, Venetoclax, Allogeneic hematopoietic stem cell transplantation, Cancer therapy, Flt3 gene, Gene mutation, Gene targeting, High risk population, Human, Idh1 gene, Idh2 gene, Myelodysplastic syndrome, Patient care, Personalized medicine, Phase 1 clinical trial (topic), Phase 2 clinical trial (topic), Review, Rna splicing

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