Zhang C, Lam SSY, Leung GMK, et al. - Researchers tested a combination treatment of sorafenib and omacetaxine mepesuccinate (SOME) among relapsed or refractory (R/R) or newly diagnosed patients with acute myeloid leukemia (AML) carrying an internal tandem duplication of Fms-like tyrosine kinase 3 (FLT3-ITD) in this phase 2 clinical trial. The participants were given sorafenib (200-400 mg twice daily) and omacetaxine mepesuccinate (2 mg daily) for 7 (first course) or 5 days (second course onward) every 21 days until progression of disease or allogeneic hematopoietic stem cell transplantation. This study included 39 R/R patients and 5 newly diagnosed patients. Complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) was achieved by 28 of the overall R/R patients, partial remission was seen in two patients, and no response in 9 patients. CR was achieved by 4 and CRi by 1 of total 5 newly diagnosed patients. The median leukemia-free survival and overall survival were estimated to be 5.6 and 10.9 months, respectively. Factors related to lower CR/CRi rates were prior FLT3 inhibitor exposure, 2 or more inductions, and coexisting IDH2 and RUNX1 mutations. Overall, findings revealed the safety as well as the efficacy of SOME for R/R and newly diagnosed FLT3-ITD AML.