Can Shorter Treatment Schedules Improve Care For Lower-Risk MDS?

Myelodysplastic syndromes (MDS) are a group of blood and bone marrow cancers. For people with lower-risk MDS, treatments should improve quality of life, reduce symptoms, and improve blood counts while minimizing side effects. A type of medication called hypomethylating agents are used to treat higher-risk and lower-risk MDS, but the same dosing schedule may not need to be the same. New research presented at the 2025 American Society of Hematology (ASH) Annual Meeting, suggests that shorter courses of hypomethylating agents may control lower-risk MDS with fewer side effects.

This study explored whether shorter dosing schedules could safely improve outcomes for patients with lower-risk MDS and chronic myelomonocytic leukemia (CMML).

What are hypomethylating agents?

Hypomethylating agents are medications that help abnormal bone marrow cells function more normally. The two most commonly used drugs are azacitidine and decitabine.

Why consider shorter treatment courses in lower-risk MDS?

Lower-risk MDS progresses more slowly, and many patients live with the condition for years. Because of this, reducing treatment intensity while maintaining effectiveness is an important goal.

Shorter treatment schedules may:

• Lower the risk of serious side effects
• Reduce time spent in clinics
• Help patients maintain independence and daily routines

A study that tested short treatment effectiveness

Researchers conducted a randomized, multicenter clinical trial to test different dosing schedules. A total of 247 patients with low- or intermediate-1-risk MDS or CMML were enrolled in the study. Patients were grouped based on whether they required blood or platelet transfusions before treatment.

Participants received one of the following:

• 3 days of decitabine, or 3 days of azacitidine, or 5 days of azacitidine
• Supportive care 

Participants were followed closely for response, safety, and long-term outcomes such as event-free survival (EFS) and overall survival (OS). EFS is the amount of time after treatment begins when a patient does not experience specific outcomes. In this study the outcomes were: 

• No response after six cycles
• Loss of response
• Increasing bone marrow blast count
• Stopping treatment because of side effects

OS is the amount of time after treatment begins that patients are still alive.

What did the study find about treatment effectiveness?

All shortened treatment schedules were generally well tolerated. However, five-day azacitidine consistently produced the strongest and most durable benefits.

Key findings included:

• Similar response rates across all drug schedules in transfusion-dependent patients.
• Around 40% to 44% of transfusion-dependent patients became transfusion-independent.
• Transfusion-independent patients had higher response rates and longer response duration when treated with five-day azacitidine.
• Five-day azacitidine showed the most consistent improvements in both EFS and OS.

Best supportive care alone led to significantly fewer responses compared with active treatment.

The study found it is safer to use shorter courses of HMA 

Shorter courses of hypomethylating agents were generally safe. Early treatment-related deaths were rare, with very low 30- and 60-day mortality rates.

Low platelet counts occurred more often with decitabine than with azacitidine. Azacitidine, especially the five-day schedule, offered a favorable balance between effectiveness and tolerability.

Five-day azacitidine stands out as the best option

Across multiple patient groups and outcome measures, five-day azacitidine provided:

• Better event-free survival
• Improved overall survival
• Longer-lasting responses
• Manageable side effects

This consistency suggests it may be the most reliable option among shortened treatment schedules for lower-risk MDS.

What does this mean for people living with lower-risk MDS?

This study supports the idea that treatment can be tailored to disease risk and personal needs. Shorter therapy schedules can reduce burden while still controlling the disease effectively.

For many patients, five-day azacitidine may offer meaningful benefits without sacrificing safety or quality of life.

If you or a loved one is living with lower-risk MDS, these findings highlight encouraging progress toward more personalized and manageable care. Treatment decisions are always individual, and ongoing conversations with your care team can help identify the approach that best supports both your health and your life beyond the clinic.

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Source: 

A randomized, multicenter trial of shorter durations of hypomethylating agents in lowerrisk Myelodysplastic Syndromes