Midostaurin for the treatment of acute myeloid leukemia.

Midostaurin is a multikinase tyrosine kinase inhibitor acting against targets known to be expressed in hematologic malignancies, especially acute myeloid leukemia. Midostaurin combined with chemotherapy followed by single-agent maintenance therapy elicited statistically significant and clinically meaningful improvement in overall survival versus placebo in patients with newly diagnosed FLT3-mutant acute myeloid leukemia. Although gastrointestinal events were more common with midostaurin, overall the drug was relatively well tolerated. Of note, midostaurin is metabolized by cytochrome P450-3A4 (CYP3A4); therefore, concomitant strong CYP3A4 inhibitors should be used with caution. Preliminary safety results from an ongoing trial evaluating midostaurin as a single agent in the post-transplant setting are encouraging. In addition, studies have evaluated its safety and efficacy in advanced systemic mastocytosis.