Impact of Frontline Treatment Strategies on Outcomes in Patients With Acute Myeloid Leukemia, Myelodysplasia-Related.

BACKGROUND

The 5th edition of the World Health Organization (WHO) classification updated the term for acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) to acute myeloid leukemia, myelodysplasia-related (AML-MR), along with new diagnostic criteria. However, the clinical prognostic outcomes of patients diagnosed with AML-MR based on the new WHO definition remain poorly defined, and the optimal frontline treatment strategy for this patient population has not been fully clarified.

AIMS: This study aimed to compare the clinical efficacy and survival outcomes of three mainstream frontline treatment regimens for newly diagnosed AML-MR patients and identify independent prognostic factors affecting patient survival.

METHODS AND

RESULTS

We collected data from 142 patients with newly diagnosed AML-MR and divided them into three cohorts depending on different frontline therapies: low-intensity chemotherapy (LIC), intensive chemotherapy (IC), and venetoclax plus hypomethylating agent (VEN/HMA). The overall response rate in the VEN/HMA cohort was higher than in the LIC or IC cohort (VEN/HMA vs. LIC, 72.7% vs. 42.1%, p = 0.010; VEN/HMA vs. IC, 72.7% vs. 54.3%, p = 0.049). Event-free survival (EFS) in the LIC cohort was inferior to that in the IC cohort (median 2.5 vs. 4.8 months, p = 0.037) or VEN/HMA cohort (median 2.5 vs. 6.4 months, p = 0.006). The overall survival (OS) in the LIC cohort was inferior to that in the IC cohort (median 11.9 vs. 19.4 months, p = 0.009). No statistically significant differences in EFS or OS were observed between the IC and VEN/HMA cohorts. Multivariate analyses showed that prior HMA exposure and frontline LIC therapy were associated with inferior EFS, and allogeneic hematopoietic stem cell transplantation was associated with better EFS and OS.

CONCLUSION

Our study suggests that in patients with AML-MR, frontline therapy with VEN/HMA or IC improves outcomes compared with LIC. Allogeneic hematopoietic stem cell transplantation is encouraged for all eligible patients with AML-MR.