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

Razan Mohty, Rama Al Hamed, Ali Bazarbachi, Eolia Brissot, Arnon Nagler, Amer Zeidan, Mohamad Mohty*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

6 Scopus citations

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.

Original languageEnglish
Article number124
JournalJournal of Hematology and Oncology
Volume15
Issue number1
DOIs
StatePublished - Dec 2022
Externally publishedYes

Funding

FundersFunder number
National Center for Advancing Translational SciencesUL1TR001863

    Keywords

    • High-risk MDS
    • Immune dysregulation
    • Immunotherapy
    • Myeloid malignancy
    • Precision medicine
    • Targeted therapy

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