Low-risk MDS: Non-transplant therapeutic approach

Moshe Mittelman*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


A significant progress has been made over the last couple of decades in understanding the biology and treatment of myelodysplastic syndromes. Based on several parameters (% blasts, cytogenteics, number of affected lineages) the patients are classified as having a lower-risk (LR) or higher risk disease. Here, we will focus on LR-MDS. The patients with LR-MDS are treated with RBC transfusions as needed, with or without erythroid stimulating agents. Luspatercept, an activin analogue, is a reasonable second line agent. Among the investigational agents in this field we can mention ruxodustat (a HIF inhibitor) and imetelstat, a telomerase inhibitor. Treatment of thrombocytopenia remain challenging and an open question.

Original languageEnglish
Pages (from-to)7-16
Number of pages10
JournalCellular Therapy and Transplantation
Issue number2
StatePublished - 2021


  • Diagnostics
  • Low-risk
  • Management
  • Myelodysplastic syndrome
  • Targeted therapy


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