Timing is Everything: New Research Might Change MDS Treatment Approach

Hypomethylating agents and stem cell transplantation are standard therapies in the care of patients with myelodysplastic syndromes (MDS) but there are key questions about the real impact on survival of these strategies. 

This study, presented at ASH 2024, looked at over 700 MDS patients to answer: How important is it for patients to show a good response to HMA therapy? Does this response matter differently for patients who eventually get a transplant versus those who don't? And for patients who do respond well to treatment, when is the best time to proceed with a transplant?

The findings are particularly valuable because they can help you and your care team make better decisions about timing and treatment approaches to get the best outcomes.

We had the privilege of interviewing the lead presenter and investigator at the 66th annual ASH conference, Dr. Benjamin Rolles, who shared insights into his research. 

Breaking Down the Findings: What Patients Need to Know

This study analyzed 762 patients with high-risk MDS from an international database, treated with hypomethylating agent-based therapies to clarify the relationship between HMA therapy, transplantation timing, and survival outcomes.
Patients were grouped into risk categories based on the IPSS-M: low, moderate, or very high risk. Responses to therapy were assessed using the IWG 2023 criteria to determine whether therapies helped achieve a composite complete response (cCR). 

What the Study Revealed

1. For patients who won't have a transplant, how well they respond to the medication really matters.

Median overall survival (OS) was significantly better in patients who achieved composite complete response and did not undergo transplantation, across all risk groups:

• Low risk: 23.5 months 
• Moderate risk: 20.8 months 
• Very high risk: 13.8 months 

However, only 37% of patients achieved a composite complete response with hypomethylating agent (HMA) therapy. This confirms that for these patients, it's very important to monitor how well the treatment is working and possibly consider changing approaches if it's not working well.

2. For patients who will have a transplant, treatment response didn’t affect long-term survival. 

For patients who were going to have a bone marrow transplant, whether or not they responded well to medication beforehand didn't make a big difference in their long-term survival. This is important because it means doctors might not need to wait for the medication to work perfectly before moving forward with a transplant.

3. Timing matters for patients who will undergo a transplant. 

Patients who received allo-HCT within the optimal window, as determined by their age and IPSS-M risk, experienced improved survival outcomes. Those transplanted at the optimal time had significantly better overall survival.

What’s Next?

This study highlights the complex relationship between treatment response, transplantation, and survival in MDS. The findings suggest that while achieving a composite complete response improves survival for patients who do not undergo transplantation, it does not independently affect survival for those who have the option to receive an allo-HCT. Instead, the timing of the transplant plays a critical role.

If you or a loved one has MDS, these findings suggest:

• Don't delay transplant decisions while waiting for medication to work perfectly
• If you're eligible for transplant, discuss timing with your healthcare team early
• If transplant isn't part of your treatment plan, how well you respond to medication therapy is very important
• Work with your healthcare team to develop a personalized treatment timeline

Conclusion

This research helps doctors make more informed decisions about when to use different treatment strategies. For patients, it emphasizes the importance of timing and personalized treatment approaches. While every patient's journey is different, these findings give both doctors and patients clearer guidance on making treatment decisions.

If you want to keep learning more MDS updates and comprehensive research insights, you can visit the HealthTree News site and bookmark it so you don’t miss out on any article! 

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Source: 

• 664 Impact of Response to Hypomethylating Agent-Based Therapy on Survival Outcomes in the Context of Baseline Clinical-Molecular Risk and Transplant Status in Patients with Myelodysplastic Syndromes/Neoplasms (MDS): An Analysis from the International Consortium for MDS (icMDS) Validate Database