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First Report of Sorafenib in Patients With Acute Myeloid Leukemia Harboring Non-Canonical FLT3 Mutations

Naval Daver1, Allyson Price1, Christopher B Benton1

  • 1The Department of Leukemia, MD Anderson Cancer Center, Houston, TX, United States.

Frontiers in Oncology
|September 28, 2020
PubMed

Insights

Targeted therapy shows promise for acute myeloid leukemia patients with rare FLT3 mutations. This study reports successful treatment using tyrosine kinase inhibitors in two cases.

Area of Science:

  • Hematology
  • Oncology
  • Molecular Biology

Background:

  • Acute myeloid leukemia (AML) prognosis is influenced by genetic mutations.
  • FLT3 mutations are common in AML, but non-canonical alterations lack prognostic data.
  • Tyrosine kinase inhibitor (TKI) use in this specific AML subgroup is unreported.

Observation:

  • Two patients with acute myeloid leukemia (AML) presented with non-canonical FLT3 mutations.
  • These mutations were neither internal tandem duplications (ITD) nor D835 point mutations.
  • Standard prognostic implications for these specific FLT3 alterations were unknown.

Findings:

  • Successful targeted therapy was achieved in both AML patients.
  • Treatment involved tyrosine kinase inhibitors directed against the non-canonical FLT3 driver alterations.
  • This represents the first reported use of TKIs in this patient population.

Implications:

  • Targeted therapy can be effective for AML with rare FLT3 mutations.
  • This approach may offer new treatment avenues for patients lacking standard therapeutic options.
  • Further research into FLT3-targeted therapies for AML is warranted.
Keywords:
AMLFLT3 mutationFLT3-N676FLT3-V592TKIsorafenib

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