Oncology Learning NetworkFLT3 Inhibitor Monotherapy Shows Promise as Strategy for Patients with High-Risk FLT3-Mutated AML

FLT3 Inhibitor Monotherapy: A Potential Pre-emptive Intervention for High-Risk FLT3-Mutated Acute Myeloid Leukemia (AML)

A recent study published in Leukemia has shed light on the potential of FLT3 inhibitor monotherapy as a pre-emptive intervention strategy for patients with high-risk FLT3-mutated acute myeloid leukemia (AML). Administered at the stage of molecular failure, characterized by high or rising levels of minimal residual disease (MRD), the therapy has shown encouraging survival rates, suggesting its potential value and the need for further exploration in this area.

Study Design

• The study focused on FLT3 inhibitor monotherapy for patients with high-risk FLT3-mutated AML.
• A total of 56 patients were included in the study, with various types of FLT3 mutations.
• More than 50% of the patients had previously been treated with midostaurin.
• All patients received FLT3 inhibitors, including gilteritinib, quizartinib, and sorafenib after molecular failure.

Key Findings

• Molecular failure was characterized by high or rising levels of MRD at a median of 9.2 months after AML diagnosis.
• 60% of patients achieved a molecular response, with 45% reaching MRD negativity.
• The patients experienced minimal hematological toxicity, which allowed 22 patients to be directly bridged to allogeneic transplantation, and another 6 to undergo donor lymphocyte infusion.
• The 2-year overall survival rate was 80% (95% confidence interval [CI], 69 to 93), and molecular event-free survival was 56% (95% CI, 44 to 72) at the 2-year mark.
• High-sensitivity next-generation sequencing for FLT3-ITD mutations at the time of molecular failure helped identify patients significantly more likely to benefit from FLT3 monotherapy.

According to the American Cancer Society, about 21,000 people get AML each year in the US, with around 10,600 dying from it.

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