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Published on: September 20, 2016
The importance of FLT3 mutational analysis in acute myeloid leukemia
1a Division of Hematology , Mayo Clinic , Rochester , MN , USA.
Abstract:
Activating mutations in FMS-like tyrosine kinase 3 (FLT3), including internal tandem duplications (ITDs) and tyrosine kinase domain (TKD) mutations, are common in patients with acute myeloid leukemia (AML). FLT3-ITD is a negative prognostic factor that remains prognostically relevant even after intensive chemotherapy and/or stem cell transplant. FLT3 testing was historically viewed as being purely prognostic; however, with the advent of FLT3 inhibitors, it will likely be seen as both prognostic and predictive. The multikinase inhibitor midostaurin, in combination with chemotherapy, is the first targeted agent to significantly prolong survival in patients with newly diagnosed FLT3-mutated AML and was recently approved by health authorities. Recently, the European LeukemiaNet recommended FLT3 testing (both TKD and ITD) for all patients with AML, with results required within 3 days. The need for optimized, multigene platform testing incorporating FLT3 mutations will increase as knowledge of interactions between FLT3 and other myeloid-relevant mutations grows.
Insights
Activating mutations in FMS-like tyrosine kinase 3 (FLT3) impact acute myeloid leukemia (AML) prognosis. FLT3 testing is now crucial for both prognosis and predicting response to targeted therapies like midostaurin.
Area of Science:
- Hematology
- Oncology
- Molecular Biology
Background:
- Activating mutations in FMS-like tyrosine kinase 3 (FLT3), including internal tandem duplications (ITDs) and tyrosine kinase domain (TKD) mutations, are frequently observed in acute myeloid leukemia (AML) patients.
- FLT3-ITD mutations represent a significant negative prognostic factor in AML, retaining their prognostic relevance despite intensive treatments like chemotherapy and stem cell transplantation.
Purpose of the Study:
- To highlight the evolving role of FLT3 testing from purely prognostic to both prognostic and predictive, driven by the development of FLT3 inhibitors.
- To emphasize the clinical significance of midostaurin as the first targeted therapy to improve survival in newly diagnosed FLT3-mutated AML.
- To underscore the European LeukemiaNet's recommendation for timely FLT3 testing in all AML patients.
Main Methods:
- Review of current literature and clinical guidelines regarding FLT3 mutations in AML.
- Analysis of the impact of FLT3 inhibitors, such as midostaurin, on patient survival.
- Discussion of the evolving diagnostic landscape for FLT3 mutation testing.
Main Results:
- Midostaurin, in combination with chemotherapy, has demonstrated significant survival prolongation in FLT3-mutated AML patients.
- FLT3 testing is increasingly recognized as predictive, guiding targeted therapy selection.
- There is a growing need for rapid, multigene testing platforms that include FLT3 mutations.
Conclusions:
- FLT3 testing is essential for all AML patients, providing both prognostic and predictive information.
- Targeted therapies like midostaurin are transforming AML treatment paradigms.
- Advancements in molecular diagnostics are crucial for optimizing personalized AML therapy.
