Sorafenib as monotherapy or in association with cytarabine and clofarabine for the treatment of relapsed/refractory FLT3 ITD-positive advanced acute myeloid leukemia

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. © 2014 Elsevier Inc. All rights reserved.