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Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome

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Published on: October 3, 2018

Updates in low/intermediate-risk MDS.

Hetty E Carraway¹

¹Cleveland Clinic, Leukemia Program, Taussig Cancer Institute, Cleveland, OH.

Hematology. American Society of Hematology. Education Program

|December 5, 2025

Summary

New therapies like luspatercept and imetelstat help manage red blood cell transfusions in low-risk myelodysplastic syndromes (LR-MDS). While these treatments offer benefits, LR-MDS can still progress to higher-risk disease.

Area of Science:

Hematology
Oncology
Molecular Biology

Background:

Low- and intermediate-risk myelodysplastic syndromes (LR-MDS/Int-MDS) involve ineffective blood cell production and specific diagnostic criteria.
Mutational profiling aids in classifying MDS, predicting prognosis, and guiding treatment decisions.

Purpose of the Study:

To review current and emerging therapeutic strategies for LR-MDS.
To discuss the management of cytopenias and disease progression in LR-MDS.

Main Methods:

Literature review of recent advancements in LR-MDS treatment.
Analysis of therapeutic options including novel agents, supportive care, and targeted therapies.

Main Results:

New agents like luspatercept and imetelstat reduce transfusion burden.

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Erythropoiesis-stimulating agents and lenalidomide are options for anemia, with potential benefits from early initiation.

Thrombopoietin receptor agonists, immunosuppressive therapy, and targeted agents for IDH mutations are also discussed.

Conclusions:

While current therapies improve symptom control and hematologic parameters in LR-MDS, the disease can evolve to higher-risk MDS or acute myeloid leukemia.
Emerging treatments offer improved management of transfusion dependence and cytopenias.