Real-Life Management of FLT3-Mutated AML: Single-Centre Experience over 24 Years.

Area of Science:

• Hematology
• Oncology
• Clinical Research

Background:

• Acute myeloid leukemia (AML) is a heterogeneous hematologic malignancy.
• Treatment strategies and prognostic factors for AML are continually evolving.
• Understanding patient subgroups and treatment responses is crucial for improving outcomes.

Purpose of the Study:

• To analyze treatment outcomes in AML patients based on mutations and treatment regimens.
• To evaluate the role of specific mutations, such as NPM1 and FLT3, in treatment response.
• To assess the impact of different stem cell transplantation approaches on survival and relapse in AML.

Main Methods:

• Retrospective analysis of 140 AML patients.
• Comparison of chemotherapy versus 3+7+midostaurin regimens.
• Evaluation of outcomes including complete remission (CR) rates, event-free survival (EFS), non-relapse mortality (NRM), and cumulative incidence of relapse (CIR).
• Analysis of prognostic factors including NPM1 mutation and white blood cell (WBC) count.

Main Results:

• Overall CR rate was 64%, higher in NPM1 mutant patients (73%).
• NPM1 mutation and lower WBC counts (< 100 × 10^9/L) positively influenced response.
• Allogeneic stem cell transplantation showed lower non-relapse mortality (28%) compared to autologous transplants (0%) but higher CIR.
• Post-transplant FLT3 inhibitors (FLT3i) showed promising survival outcomes in a subset of patients.

Conclusions:

• NPM1 mutation and WBC count are significant prognostic factors in AML.
• Allogeneic transplantation is critical for FLT3-mutated AML, necessitating careful patient selection for CR1.
• Further research is needed to optimize post-transplant strategies and identify patients who benefit most from intensified therapy.