(mye doe STAW rin)
Trade Name(s): Rydapt®
Midostaurin is the generic name for the trade name drug Rydapt. In some cases, health care professionals may use the trade name Rydapt when referring to the generic drug name midostaurin.
Drug Type: Midostaurin is a targeted anti-cancer therapy. This medication is classified as a tyrosine kinase inhibitor, more specifically a FLT3 inhibitor. (For more detail, see "How Midostaurin Works" below)
What Midostaurin Is Used For:
- Acute Myeloid Leukemia (AML) that is FLT3-positive
- Mast Cell Leukemia (MCL)
- Aggressive Systemic Mastocytosis (ASM)
- Systemic Mastocytosis with Associated Hematological Neoplasm (SM-AHN)
Note: If a drug has been approved for one use, physicians may elect to use this same drug for other problems if they believe it may be helpful.
How Midostaurin Is Given
- This drug is given by mouth with food
- Anti-nausea medications may be given prior to midostaurin
- Doses are 50 mg to 100 mg twice daily
The amount of midostaurin and duration of therapy that you will receive depends on many factors, including your general health or other health problems, the type of cancer or condition you have, and toxicities experienced while on midostaurin. Your doctor will determine your exact dosage and schedule.
Important things to remember about the side effects of midostaurin:
- Most people will not experience all of the midostaurin side effects listed.
- Midostaurin side effects are often predictable in terms of their onset, duration, and severity.
- Midostaurin side effects will improve after therapy is complete.
- Midostaurin side effects may be quite manageable. There are many options to minimize or prevent the side effects of midostaurin.
The following side effects are common (occurring in greater than 30%) for patients taking midostaurin for acute myeloid leukemia (AML):
The following side effects are common (occurring in greater than 30%) for patients taking midostaurin for Mast Cell Leukemia (MCL), Aggressive Systemic Mastocytosis (ASM), or Systemic Mastocytosis with Associated Hematological Neoplasm (SM-AHN):
These are less common side effects (occurring in 10-29%) for patients receiving midostaurin:
Not all side effects are listed above. Side effects that are very rare - occurring in less than about 10 percent of patients - are not listed here. But you should always inform your health care provider if you experience any unusual symptoms.
When To Contact Your Doctor or Health Care Provider
Contact your health care provider immediately, day or night, if you should experience any of the following symptoms:
- Fever of 100.4º F (38º C) or higher, chills (possible signs of infection)
- Shortness of breath, chest discomfort, or non-productive cough, as these are symptoms of lung disease. Fatal cases of lung disease, or pulmonary toxicity, have occurred with midostaurin.
The following symptoms require medical attention, but are not an emergency. Contact your health care provider within 24 hours of noticing any of the following:
- Nausea (interferes with ability to eat and unrelieved with prescribed medication)
- Vomiting (vomiting more than 4-5 times in a 24 hour period)
- Diarrhea (4-6 episodes in a 24-hour period)
- Unusual bleeding or bruising
- Black or tarry stools, or blood in your stools
- Blood in the urine
- Pain or burning with urination
- Extreme fatigue (unable to carry on self-care activities)
- Mouth sores (painful redness, swelling or ulcers)
Always inform your health care provider if you experience any unusual symptoms.
- Before starting midostaurin treatment, make sure you tell your doctor about any other medications you are taking (including prescription, over-the-counter, vitamins, herbal remedies, etc.). Do not take aspirin, products containing aspirin unless your doctor specifically permits this.
- Other medications that should not be taken with midostaurin are amiodarone, citalopram, and fluoxetine, as they can prolong the QT-interval, which can cause life-threatening arrhythmias.
- Medications such as certain azole anti-fungals (itraconazole, voriconazole, posaconazole), clarithromycin, diltiazem, and certain anti-virals (atazanavir, ritonavir) can cause increased the effects of midostaurin leading to toxicity
- Medications such as phenobarbital, phenytoin, carbamazepine, and St. John's Wort should also not be taken with midostaurin, as they can decrease how well it works.
- Do not receive any kind of immunization or vaccination without your doctor's approval while taking midostaurin.
- Inform your health care professional if you are pregnant or may be pregnant prior to starting this treatment. Animal reproduction studies have shown adverse effects at midostaurin doses lower than those used in humans. Midostaurin may cause fetal harm if used in pregnant women.
- For both men and women: Use contraceptives, and do not conceive a child (get pregnant) while taking midostaurin. Barrier methods of contraception, such as condoms, are recommended for up to 4 months after last dose of midostaurin.
- Do not breast feed while taking and for at least 4 months after the last dose of midostaurin.
- If you vomit after taking a dose of midostaurin, do not take an extra dose. Take your next dose at your scheduled time
- Take midostaurin with food; do not open or crush the capsules
- You may be at risk of infection so try to avoid crowds or people with colds, and report fever or any other signs of infection immediately to your health care provider.
- Wash your hands often.
- To help treat/prevent mouth sores, use a soft toothbrush, and rinse three times a day with 1 teaspoon of baking soda mixed with 8 ounces of water.
- Use an electric razor and a soft toothbrush to minimize bleeding.
- To reduce nausea, take anti-nausea medications as prescribed by your doctor, and eat small, frequent meals.
- In general, drinking alcoholic beverages should be kept to a minimum or avoided completely. You should discuss this with your doctor.
- Get plenty of rest.
- Maintain good nutrition.
- Remain active as you are able. Gentle exercise is encouraged such as a daily walk.
- If you experience symptoms or side effects, be sure to discuss them with your health care team. They can prescribe medications and/or offer other suggestions that are effective in managing such problems.
Monitoring and Testing While Taking Midostaurin
You will be checked regularly by your doctor while you are taking midostaurin, to monitor side effects and check your response to therapy. Periodic blood work will be obtained to monitor your complete blood count (CBC) as well as the function of other organs (such as your kidney and liver) will also be ordered by your doctor. An EKG will be checked for QTc prolongation. If you are a woman of childbearing age, you will have a pregnancy test within 7 days before starting midostaurin.
How Midostaurin Works
Targeted therapy is the result of about 100 years of research dedicated to understanding the differences between cancer cells and normal cells. To date, cancer treatment has focused primarily on killing rapidly dividing cells because one feature of cancer cells is that they divide rapidly. Unfortunately, some of our normal cells divide rapidly too, causing multiple side effects.
Targeted therapy is about identifying other features of cancer cells. Scientists look for specific differences in the cancer cells and the normal cells. This information is used to create a targeted therapy to attack the cancer cells without damaging the normal cells, thus leading to fewer side effects. Each type of targeted therapy works a little bit differently but all interfere with the ability of the cancer cell to grow, divide, repair and/or communicate with other cells.
There are different types of targeted therapies, defined in three broad categories. Some targeted therapies focus on the internal components and function of the cancer cell. The targeted therapies use small molecules that can get into the cell and disrupt the function of the cells, causing them to die. There are several types of targeted therapy that focus on the inner parts of the cells. Other targeted therapies target receptors that are on the outside of the cell. Therapies that target receptors are also known as monoclonal antibodies. Antiangiogenesis inhibitors target the blood vessels that supply oxygen to the cells, ultimately causing the cells to starve.
Researchers agree that targeted therapies are not a replacement for traditional therapies. They may be best used in combination with traditional therapies. More research is needed to identify which cancers may be best treated with targeted therapies and to identify additional targets for more types of cancer.
Midostaurin is a targeted therapy that targets and inhibits tyrosine kinases on the surface of the cell. FLT3 is a tyrosine kinase that can stimulate leukemic cell production. By binding to these receptors midostaurin blocks an important pathway that promotes cell division and causes death to leukemic cells.
Note: We strongly encourage you to talk with your health care professional about your specific medical condition and treatments. The information contained in this website is meant to be helpful and educational, but is not a substitute for medical advice.