ASH 2025: Stem Cell Transplant Outcomes in MDS Have Improved Over the Last 20 Years

Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only approved curative treatment for myelodysplastic syndromes (MDS). It is usually offered to people with higher-risk disease because it also carries some risks, and people need to be matched to a suitable donor to avoid complications.

At the American Society of Hematology (ASH) meeting in December, researchers presented an important analysis from nearly 19,000 people with MDS who received a transplant over the past 20 years. The goal was to understand whether transplant results have improved over time and which factors most affect survival.

An analysis of over 20 years tracked changes across decades in people who received allogeneic transplant.

This was a large registry-based study using data from the European Society for Blood and Marrow Transplantation (EBMT). A total of 18,710 adults with MDS who received an allogeneic transplant between 2000 and 2022 were included. The analysis focused mainly on these four aspects:

• Overall survival (OS): how long patients lived after transplant
• Relapse-free survival (RFS): time without disease returning
• Non-relapse mortality (NRM): deaths from transplant-related causes
• Relapse: return of MDS after transplant

More older and higher-risk patients are now undergoing transplant than in the past

Across 20 years, the median age at transplant increased from about 50 years to over 61 years. Other important findings indicate meaningful progress in transplant safety and effectiveness: 

• Most patients had MDS with excess blasts, a higher-risk subtype
• Transplants came from diverse donors, including matched and mismatched family or unrelated donors, as well as cord blood
• Overall survival improved from about 44% in the early 2000s to over 50% in recent years
• Relapse rates stayed stable, suggesting better supportive care rather than reduced disease risk, and non-relapse mortality dropped significantly, meaning fewer deaths from transplant complications

What factors affected transplant outcomes?

Donor type was crucial because researchers noted that outcomes were best with fully matched related donors. Other factors also had an impact on outcomes:

• Age at transplant
• Physical function and other chronic diseases burden
• Cytogenetic risk
• Being transplanted when MDS was not in remission

How have transplant approaches changed?

Reduced-intensity conditioning regimens lowered the risk of transplant-related death. However, they were associated with a slightly higher chance of relapse, highlighting the need for personalized decision-making.

Importantly, even after adjusting for these factors, patients transplanted in more recent years still did better, suggesting broad advances in transplant platforms, infection prevention, and complication management.

What does this mean for people with MDS?

This study offers encouraging news. Stem cell transplantation for MDS has become safer and more effective, even as it is offered to older and more complex patients.

For people considering transplant, this research supports:

• Earlier referral to transplant centers
• Careful evaluation of donor options
• Individualized discussion of risks and benefits

If you or a loved one is living with MDS, it’s important to know that transplant care is evolving. Advances in supportive care, donor selection, and transplant techniques are improving outcomes year by year. Talk with your care team about whether a transplant may be an option, and remember that today’s results reflect decades of learning, progress, and hope.

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Source: Trends in transplantation for patients with myelodysplastic syndrome across recent decades: An EBMT registry-based study