How it is administered

Midostaurin is taken by mouth as a soft capsule, usually with food. The typical dose for acute myeloid leukemia (AML) is 50 mg twice daily, while for aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL), the dose is 100 mg twice daily. Capsules should not be opened or crushed. 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 kinase inhibitor, which means it blocks certain enzymes (kinases) that help cancer cells grow and survive. Specifically, it targets multiple receptor tyrosine kinases, including FLT3 (wild type and mutant), KIT (wild type and D816V mutant), and PDGFRα/β. By inhibiting these kinases, midostaurin disrupts signals that promote the growth and survival of abnormal blood cells in certain blood cancers.

In acute myeloid leukemia (AML) with FLT3 mutations, midostaurin helps to inhibit the abnormal signaling that drives the rapid growth of leukemia cells. In systemic mastocytosis and related conditions, it blocks the activity of mutated KIT, which is often responsible for the uncontrolled growth of mast cells. This action can help slow disease progression and improve symptoms.

Who Should take it

Midostaurin is used for adults with:

• Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation-positive, in combination with standard chemotherapy (cytarabine and daunorubicin for induction, and cytarabine for consolidation).
• Aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

It is important that patients with AML have their FLT3 mutation status confirmed by an FDA-approved test before starting midostaurin. For mastocytosis and related diseases, it is used for advanced cases where other treatments may not be effective.

Who should not take it

You should not take midostaurin if you have had a severe allergic reaction (hypersensitivity) to midostaurin or any of its ingredients. Symptoms of hypersensitivity can include anaphylactic shock, difficulty breathing, flushing, chest pain, or swelling of the airways or tongue.

Midostaurin is not recommended for use during pregnancy, 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 female partners of childbearing potential should also use contraception. Breastfeeding is not recommended during treatment and for 4 months after the last dose.

Commonly used with

For AML, midostaurin is used in combination with standard chemotherapy agents, specifically cytarabine and daunorubicin for induction, and cytarabine for consolidation. It is not used as a single agent for induction therapy in AML.

For advanced systemic mastocytosis and related conditions, it is typically used as a single agent.

Commonly tested with

In clinical trials for AML, midostaurin was tested in combination with standard chemotherapy (cytarabine and daunorubicin). For systemic mastocytosis and related diseases, it was tested as a single agent. In some studies, it has also been evaluated with other supportive medications, such as anti-emetics to manage nausea and vomiting.