Objective: To explore the safety and efficacy of sorafenib in combination with chemotherapy for the treatment of FLT3 positive acute myeloid leukemia (AML), to highlight the impact of FLT3 mutations and targeting therapy on response of AML.
Methods: The clinical and laboratory features and the treatment response, especially the safety profile of sorafenib in an acute monocytic leukemia patient with FLT-ITD were reported.
Results: The patient achieved clinical and molecular CR after sorafenib was added to the second course of combination chemotherapy. The side effects of sorafenib were mild and tolerable.
Conclusion: The patient responded well to the combination of sorafenib and standard chemotherapy of AML without significant adverse effects.