How it Works

Midostaurin is a targeted therapy that is used to treat acute myeloid leukemia (AML) and advanced systemic mastocytosis (ASM). It is classified as a tyrosine kinase inhibitor, which means that it blocks the activity of a type of protein called a tyrosine kinase.

Tyrosine kinases are proteins that play a role in the growth and spread of cancer cells by transmitting signals within cells. When these proteins are activated, they can stimulate the growth and division of cancer cells and promote the formation of new blood vessels, which can help cancer cells to spread and grow.

Midostaurin specifically targets a protein called FLT3, which is involved in the development of AML. It blocks the activity of FLT3 by inhibiting the receptor signaling and cell proliferation of leukemic cells expressing internal tandem duplication (ITD)  and tyrosine kinase domain (TKD) mutant FLT3 receptors or overexpressing wild-type FLT3 and PDGF receptors. Midostaurin also has the ability to inhibit KIT signaling (a receptor found in some types of cancer), cell proliferation, and histamine release and induce cell death in mast cells (advanced systemic mastocytosis cells).

How it’s Administered

• Given as capsules taken by mouth. It is usually taken once a day, at the same time each day, this capsule should be swallowed whole without breaking it or crushing it. 
• You must avoid grapefruit produce as they interfere with the way the medication is metabolized.  

Who Should Take Midostaurin 

• Indicated, combined with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy, for adults with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive.
• Indicated for adults with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL)

Who Shouldn’t Take Midostaurin

• Patients that have a severe allergy to midostaurin or any of its components.
• Pregnant women. It is important to inform your healthcare provider if you are pregnant, or planning to become pregnant before starting treatment, as it may cause harm to the fetus.
• Breastfeeding women should suspend breastfeeding if they start taking this medication. 
• If taking the following medications, like azole anti-fungals, amiodarone, citalopram, and fluoxetine, as they are contraindicated while ingesting midostaurin. 

The Most Common Side Effects of Taking Midostaurin Include:

• Nausea, vomiting, inflammation of mouth tissues, hemorrhoids, bleeding of the nose  
• Increased liver enzymes, low calcium
• Musculoskeletal pain, headache, joint pain, fever
• Petechiae (pinpoint, round spots that appear on the skin as a result of bleeding, they commonly appear in clusters and may look like a rash), low platelet counts

Commonly Used in Acute Myeloid Leukemia With

• Daunorubicin and cytarabine.
• High-dose cytarabine.

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Date last updated: 12/22/22