Sorafenib As Monotherapy or in Association With Cytarabine and Clofarabine for the Treatment of Relapsed/Refractory FLT3 ITD-Positive Advanced Acute Myeloid Leukemia

Clinical Practice Points � Acute myeloid leukemia (AML) associated with fmsrelated tyrosine kinase 3 (FLT3) internal tandem duplication mutation has an increased relapse rate and a reduced overall survival. � Here we report 3 cases of advanced refractory AML treated using sorafenib alone or in association with chemotherapy, at a standard dose of 400 mg twice daily. All the patients had been previously treated with several chemotherapy lines and their status was complicated by severe infectious disease. � After sorafenib treatment, clinical complete remission but not molecular remission was achieved in all cases. Median duration of the response of these patients was relatively short (40 days), but in 1 patient it was possible to continue the therapy at the reduced dose of 600 mg daily as a bridge to allogeneic stem cell transplantation. � We confirm that sorafenib is active in FLT3 mutated AML and we suggest that this drug could be used in more precocious stages of this disease to achieve a deeper hematological response as a bridge to bone marrow transplant.