How it is administered

Midostaurin is taken by mouth as a capsule. Each capsule contains 25 mg of midostaurin. For acute myeloid leukemia (AML), the usual dose is 50 mg (two capsules) twice daily with food, on specific days of the chemotherapy cycle (Days 8 to 21 of each cycle of induction and consolidation chemotherapy). It is important to swallow the capsules whole and not to open or crush them. If a dose is missed or vomited, do not make up the dose; take the next dose at the usual scheduled time.

How it works

Midostaurin is a type of medication called a kinase inhibitor. It works by blocking the activity of certain proteins called receptor tyrosine kinases, which are involved in the growth and survival of cancer cells. In particular, midostaurin targets the FLT3 protein, which is often mutated in some patients with acute myeloid leukemia (AML). These mutations can cause leukemia cells to grow uncontrollably.

By inhibiting FLT3 and other related proteins, midostaurin helps to stop the growth and spread of leukemia cells. It also promotes the death (apoptosis) of these cancer cells. This makes midostaurin especially useful for patients whose AML tests positive for FLT3 mutations. The medication is used in combination with standard chemotherapy to improve outcomes for these patients.

Who Should take it

Midostaurin is indicated for adults with newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive, as detected by an FDA-approved test. It is used in combination with standard chemotherapy drugs (cytarabine and daunorubicin) during both induction and consolidation phases of treatment.

It is not used as a single-agent induction therapy for AML. Midostaurin may also be prescribed for certain rare blood disorders, such as aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, or mast cell leukemia, but its primary use in blood cancers is for FLT3-mutated AML.

Who should not take it

Midostaurin should not be taken by anyone who has had a hypersensitivity (allergic) reaction to midostaurin or any of its ingredients. Signs of hypersensitivity can include anaphylactic shock, difficulty breathing, flushing, chest pain, or swelling of the airways or tongue.

Women who are pregnant should not take midostaurin, as it may cause harm to an unborn baby. Women of childbearing potential should use effective contraception during treatment and for 4 months after the last dose. Men with partners who could become pregnant should also use effective contraception during and for 4 months after treatment. Breastfeeding is not recommended during treatment and for 4 months after the last dose.

Commonly used with

Midostaurin is used in combination with standard chemotherapy drugs for AML, specifically cytarabine and daunorubicin during induction and consolidation phases. It is not used alone for induction therapy in AML.

Supportive medications, such as anti-emetics (to prevent nausea and vomiting), may also be given to help manage side effects.

Commonly tested with

Midostaurin has been tested in combination with standard AML chemotherapy regimens, particularly cytarabine and daunorubicin. In clinical studies, it was compared to placebo in combination with these chemotherapy agents in patients with FLT3-mutated AML.

It has also been studied in patients with advanced systemic mastocytosis as a single agent.