Rydapt (midostaurin) is a first-choice oral medication for acute myeloid leukemia (AML) that's FLT3-positive. It's taken in combination with chemotherapy for people who are newly diagnosed with this type of AML. Rydapt (midostaurin) can also help treat some types of systemic mastocytosis, a condition where there's an overgrowth of certain cells of the immune system. Some common side effects of this medication include nausea, vomiting, and high blood sugar.
Acute myeloid leukemia (AML) with a specific mutation (FLT3-positive)
Rydapt (midostaurin) is a type of targeted therapy called a tyrosine kinase inhibitor. Kinases are enzymes (proteins) that send signals to cells, including cancer cells, that tells them to grow. Rydapt (midostaurin) works by blocking several kinases in blood cancer (leukemia) cells, which then prevents these cells from growing. Rydapt (midostaurin) works specifically on leukemia cells that have the FLT3 mutation.
For systemic mastocytosis and mast cell leukemia, Rydapt (midostaurin) is thought to work by slowing or stopping the growth of abnormal mast cells. It might also block the release of histamines that cause symptoms of systemic mastocytosis.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report to your care team if they continue or are bothersome):
Taken by mouth
First-choice treatment option for newly diagnosed FLT3-positive AML
More precise in the way it targets cancer cells, compared to traditional chemotherapy
Taken twice daily
Must take with food
Commonly causes nausea and vomiting
Take Rydapt (midostaurin) with food. Take the medication twice a day, about every 12 hours, and swallow the capsule whole. Don’t chew, crush, or break open the capsule.
Rydapt (midostaurin) should be avoided during pregnancy because it can harm the unborn baby. If you're female and able to become pregnant, your provider will ask you to take a pregnancy test a week before you start Rydapt (midostaurin). You should also take birth control while receiving Rydapt (midostaurin) treatment and for 4 months after your last dose.
If you're male and have a female partner(s) who's able to become pregnant, you should also use birth control during treatment with Rydapt (midostaurin) and for 4 months after the last dose.
If you experience any breathing problems while taking Rydapt (midostaurin), let your provider know. This medication can cause serious lung problems.
Rydapt (midostaurin) can raise your blood sugar levels. If you already have diabetes, your provider might ask you to check your blood sugar more often to see if it’s within a healthy range. Make sure to let your provider know if you have a hard time controlling your blood sugar levels because your provider might need to make changes to your blood sugar medications.
Let your provider know if you experience nausea or vomiting while taking Rydapt (midostaurin). If appropriate, they might prescribe medications to ease your symptoms. Your provider might also recommend other tips to prevent nausea and vomiting, such as eating bland foods, eating smaller meals more often, or drinking small sips of water throughout the day.
If you vomit after taking Rydapt (midostaurin) (or if you miss a dose of Rydapt (midostaurin)), don’t take an extra dose to make it up. Just skip the missed dose and take your next dose of Rydapt (midostaurin) at the next scheduled time.
Ask your provider or pharmacist about the best way to handle and throw away Rydapt (midostaurin) safely. Anticancer medications can be very harmful to people who handle or come into contact with them.
Rydapt (midostaurin) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
It's not common, but Rydapt (midostaurin) can cause serious lung problems, like inflammation of your lungs or interstitial lung disease. Let your provider if you develop any new or worsening breathing problems. Your provider might tell you to stop taking Rydapt (midostaurin).
Based on animal studies, Rydapt (midostaurin) can harm an unborn baby if taken by mothers during pregnancy. Because of this, Rydapt (midostaurin) shouldn’t be taken during pregnancy. If you’re female and able to become pregnant, your provider will ask you to do a pregnancy test a week before you start this medication. You should take birth control while taking Rydapt (midostaurin) and for at least 4 months after the last dose. Males with female partners who can become pregnant should also use birth control during treatment with Rydapt (midostaurin) and for 4 months after the last dose. Let your provider know right away if you become pregnant during this time.
AML: The typical dosing is 50 mg by mouth twice daily on Day 8 through 21 of each treatment cycle.
Mast cell leukemia and systemic mastocytosis: The typical dosing is 100 mg by mouth twice daily.
Your provider might adjust your dose if you experience certain side effects or if you’re taking medications that might interact with Rydapt (midostaurin).
Acute myeloid leukemia (AML) with a specific mutation (FLT3-positive)
Acute myeloid leukemia (AML) treatment
Acute lymphoblastic leukemia (ALL) treatment
Chronic myeloid leukemia (CML) treatment
Meningeal leukemia treatment and prevention - if given intrathecally
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National Cancer Institute. (2023). Acute myeloid leukemia treatment (PDQ®)–Patient version.
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Novartis Pharmaceuticals Corporation. (n.d.). Understanding treatment.
Novartis Pharmaceuticals Corporation. (2023). Rydapt capsule, liquid filled [package insert]. DailyMed.
Novartis Pharmaceuticals Corporation. (2023). Rydapt® (midostaurin) capsules, for oral use [package insert].
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