How Can Hypomethylating Agents Improve Platelet Counts in MDS?

Low platelet counts, also called thrombocytopenia, are common in myelodysplastic syndromes (MDS) and can increase the risk of bruising and bleeding. New research presented at the 2025 American Society of Hematology (ASH) meeting helps explain why some patients see platelet improvements early in treatment with hypomethylating agents (HMAs), and who may benefit the most.

This study offers encouraging insights into how these treatments work at a cellular level. 

The role of platelets and hypomethylating agents (HMAs) in MDS

Platelets aid wound healing and help prevent bleeding. In MDS, the bone marrow does not make healthy blood cells effectively, which often leads to low platelet counts. Having low platelet counts can limit MDS treatment options.

Hypomethylating agents are a type of drug commonly used as first-line treatment for MDS. The most widely used HMAs include azacitidine and decitabine. Some people experience a rise in platelet counts early in HMA treatment, and this early improvement is often associated with better overall outcomes.

A specific gene mutation has larger benefits from HMAs in platelets 

In this study, researchers analyzed platelet counts from 319 patients with MDS treated with HMAs. They found that patients who started treatment with very low platelet counts benefited the most.

There was also observed in people with a TET2 gene mutation a much larger increase in platelets after two cycles of treatment compared with patients without this mutation.

The importance of understanding HMA benefits for MDS patients 

Understanding how HMAs improve platelet counts helps doctors better predict who may benefit from treatment. It also opens the door to developing new therapies specifically designed to support platelet recovery.

For patients with very low platelet levels and those with TET2 mutations, these findings offer reassurance that early platelet improvements are meaningful for better outcomes.

Research is not always about finding new treatments. It also helps us learn the best ways we could benefit from existing ones, and long-term outcomes 

Keep reading more research news, treatment advances and patient stories with the healthtree for MDS newsletter, where we compile the most relevant information for patients and caregivers. 

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Source:

•  Hypomethylation agent improves late-stage megakaryocyte maturation in MDS