Sorafenib for refractory FMS-like tyrosine kinase receptor-3 (FLT3/ITD+) acute myeloid leukemia after allogenic stem cell transplantation.

Mutations of FLT3 in AML is an entity characterized by a significant poor prognosis and are described in about 30% of AML in adults. Usually patients with this subtype of AML carrying either internal tandem duplication (ITD) or mutation at codon 835 are referred for allogeneic stem cell transplantation (ASCT) but a proportion of them would eventually relapse. The introduction of targeted therapy against FLT3 + AML is yielding conflicting results and many trials are running with new molecules. We used sorafenib, a multi-targeted tyrosine kinase inhibitor FDA approved for the treatment of advanced renal cell and hepatocellular carcinoma, on a compassionate basis in two patients with FLT3 + AML relapsing after ASCT.