Midostaurin for the management of FLT3-mutated acute myeloid leukemia and advanced systemic mastocytosis.

Purpose: This article reviews the pharmacology, efficacy, safety, cost, and future directions of midostaurin for the treatment of acute myeloid leukemia (AML), aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, and mast cell leukemia, collectively known as advanced systemic mastocytosis (SM). Summary: Midostaurin was approved by the U.S. Food and Drug Administration for the treatment of FMS-like tyrosine kinase-3 (FLT3)- mutated AML. FLT3 is a tyrosine kinase which plays a key role in proliferation of early hematopoietic progenitor cells. In addition to having activity against FLT3, midostaurin inhibits several other tyrosine kinases which led to its approval for the treatment of advanced SM.

Conclusion: Midostaurin offers a novel strategy to treat both FLT3-mutated AML and advanced SM. With a comparable adverse effect profile to other agents and substantial antiproliferative activity, midostaurin offers a therapeutic option for patients who have historically been difficult to treat.