Home›Hematology› Treosulfan-fludarabine improves 24-month event-free survival to 65% versus 53% in AML patients
Treosulfan-fludarabine improves 24-month event-free survival to 65% versus 53% in AML patientsTreosulfan Shows Better Outcomes for Patients with Acute Myeloid Leukemia
HaematologicaPublished September 1, 2026Study authors: Stölzel Friedrich, Stelljes Matthias, Beelen Dietrich Wilhelm, Markiewicz Miroslaw, Reményi Péter, L…PubMed ↗DOI ↗Editorial oversight: Dr. Amelia Tan, PhD · Internal Medicine & Chronic Disease
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Key Takeaway
Consider treosulfan-fludarabine as a more effective and safer conditioning regimen for AML patients undergoing alloHCT.
This Phase 3 randomized trial subgroup analysis included 352 patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic cell transplantation (alloHCT). The study compared a treosulfan-fludarabine conditioning regimen against a reduced-intensity busulfan-fludarabine regimen.
Primary outcomes showed treosulfan-fludarabine superiority for 24-month event-free survival (65% vs. 53%, P=0.01). In patients with HCTCI score >2, the 24-month event-free survival was 62% for treosulfan-fludarabine versus 42% for busulfan-fludarabine (P=0.02). Secondary outcomes included overall survival of 73% vs. 65% for treosulfan-fludarabine and GvHD-free and relapse-free survival of 53% vs. 40% (P=0.02).
Regarding safety, the treosulfan-fludarabine regimen was reported to have a more favorable safety profile. Specifically, the incidence of extensive chronic GvHD at 24 months was 15.1% for treosulfan-fludarabine compared to 28.1% for busulfan-fludarabine (P=0.01).
A primary limitation of these findings is that the data presented are from a subgroup analysis. While treosulfan-fludarabine appears more effective and safer for AML patients undergoing alloHCT, particularly those at higher risk, clinicians should interpret these results with the caution appropriate for subgroup data.
How this fits prior evidence
How this fits prior evidence: This finding addresses a gap in conditioning regimen options for AML patients undergoing alloHCT. While prior coverage noted that MRG mutations correlate with worse overall survival and lower remission in NPM1-mutated AML, this study provides specific data on treosulfan-fludarabine as a more effective and safer conditioning regimen for AML patients, particularly those at higher risk.
This study looked at patients with acute myeloid leukemia (AML) who were undergoing a specific type of stem cell transplant called allogeneic hematopoietic cell transplantation. Researchers compared two different conditioning methods: a treosulfan-fludarabine mix and a busulfan-fludarabine mix. The goal was to see which method led to better survival and fewer complications.
The results showed that patients who received the treosulfan-fludarabine regimen had a higher 24-month event-free survival rate of 65% compared to 53% for those on the busulfan-fludarabine regimen. Additionally, the treosulfan group saw a higher overall survival rate of 73% compared to 65%. The study also found that the treosulfan group had a lower rate of severe chronic graft-versus-host disease at 24 months.
It is important to note that these specific findings come from a subgroup analysis of a larger Phase 3 trial. While the treosulfan regimen appeared more effective and safer in this study, the results are based on a specific subset of patients. You should talk to your doctor to see how these findings apply to your specific treatment plan.
What this means for you:
Treosulfan may improve survival and reduce complications for some patients with acute myeloid leukemia.
Common questions
Is treosulfan safer than busulfan for leukemia patients?
The study found that the treosulfan-fludarabine regimen had a more favorable safety profile compared to the busulfan-fludarabine regimen. Specifically, the treosulfan group had a lower incidence of extensive chronic graft-versus-host disease at 24 months, with 15.1% compared to 28.1% in the busulfan group.
How did the survival rates compare between the two treatments?
Patients receiving treosulfan-fludarabine showed a 65% 24-month event-free survival rate, while those receiving busulfan-fludarabine had a 53% rate. The overall survival rate was also higher for the treosulfan group at 73% compared to 65% for the busulfan group.
Who specifically benefited from the treosulfan treatment?
The study showed significant benefits for patients with a higher risk score (HCTCI score >2). In this specific group, the 24-month event-free survival was 62% for those receiving treosulfan compared to 42% for those receiving busulfan.
Acute myeloid leukemia (AML) is the most common indication for allogeneic hematopoietic cell transplantation (alloHCT), yet graft-versus-host disease (GvHD) remains a major post-transplant complication. Conditioning regimens, particularly reduced-intensity approaches, are critical in optimizing outcomes. This subgroup analysis of the phase III MC-FludT.14/L trial compared treosulfan-fludarabine with reduced-intensity busulfan-fludarabine in 352 AML patients (aged 31-70 years) undergoing alloHCT. The primary endpoint was 24-month event-free survival; secondary endpoints included overall survival, GvHD incidence, relapse/progression, and non-relapse mortality. Treosulfan compared to busulfan demonstrated superiority: 24-month event-free survival was 65% vs. 53% (P=0.01), and overall survival was 73% vs. 65%. Event-free survival benefits were consistent across AML risk categories and notably higher in patients with Hematopoietic Cell Transplantation Comorbidity Index score >2 (62% vs. 42%, P=0.02). Treosulfan also showed lower non-relapse mortality and relapse rates. GvHD outcomes favored treosulfan, with a significantly lower incidence of extensive chronic GvHD at 24 months (15.1% vs. 28.1%, P=0.01). GvHD-free and relapse-free survival was also improved (53% vs. 40%, P=0.02). The safety profile was more favorable with treosulfan. These findings support treosulfan-fludarabine as a more effective and safer conditioning regimen than busulfan-fludarabine for AML patients undergoing alloHCT, particularly those at higher risk.
