Midostaurin for the treatment of acute myeloid leukemia

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7 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1853-1871
Number of pages19
JournalFuture Oncology
Issue number21
StatePublished - Sep 2017


  • FLT3
  • PKC412
  • acute myeloid leukemia
  • allogeneic hematopoietic stem cell transplant
  • midostaurin
  • tyrosine kinase inhibitor

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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