Midostaurin – Uses, Dosage, Side Effects, Interaction

Midostaurin - Uses, Dosage, Side Effects, Interaction
Patient Tools

Read, save, and share this guide

Use these quick tools to make this medical article easier to read, print, save, or share with a family member.

On this page13 sections

Article Summary

Midostaurin is an orally available small molecule inhibitor of FMS-like tyrosine kinase 3 (FLT3) which is used as an antineoplastic agent in the treatment of acute myeloid leukemia with FLT3 mutations. Midostaurin is associated with a moderate rate of serum aminotransferase elevations during therapy and is suspected to cause rare instances of clinically apparent acute liver injury. Midostaurin is a synthetic indolocarbazole multikinase inhibitor with potential antiangiogenic...

Key Takeaways

  • This article explains Mechanism of Action in simple medical language.
  • This article explains Indications in simple medical language.
  • This article explains Contraindications in simple medical language.
  • This article explains Dosage in simple medical language.
Before reading

RX Patient Tools

Use these quick guides before reading the article, or return to them when you need help preparing questions for a doctor.

Start here Choose the right pathway for symptoms, reports, medicines, or urgent warning signs. Disease article roadmap Read this topic step by step: meaning, symptoms, warning signs, diagnosis, treatment, prevention, and follow-up. Treatment planner Prepare questions about treatment choices, benefits, risks, side effects, and follow-up. Family & caregiver guide Organize symptoms, reports, medicines, questions, and follow-up safely. Nutrition & diet guide Prepare food, hydration, supplement, and medicine-timing questions safely. Prevention guide Organize risk factors, protective habits, screening, and warning signs. Recovery guide Prepare a safe plan for activity, rehabilitation, warning signs, and follow-up.
Educational health guideWritten for patient understanding and clinical awareness.
Reviewed content workflowUse writer and reviewer profiles for stronger trust.
Emergency safety firstUrgent warning signs are highlighted below.
Choose your reading view

Patient View highlights a simple learning journey. Clinical View reveals structure, evidence, and editorial completeness.

Indications

  • Investigated for use/treatment in adult patients with high-risk acute myeloid leukemia (AML) who are FLT3 mutation-positive, aggressive mastocytosis (ASM), systemic mastocytosis with associated hematological (SM-AHN), or mast cell leukemia (MCL).
  • Midostaurin is an antineoplastic agent used to treat high-risk acute myeloid leukemia (AML) with specific mutations, aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell leukemia (MCL).
  • Rydapt is indicated: in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM AHN), or mast cell leukemia (MCL).
  • Midostaurin is an orally available small molecule inhibitor of FMS-like tyrosine kinase 3 (FLT3) which is used as an antineoplastic agent in the treatment of acute myeloid leukemia with FLT3 mutations.
  • In combination with standard cytarabine and daunorubicin induction and cytarabine consolidation
    chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are FLT3
    mutation-positive, as detected by an FDA-approved test.
  • Acute Myeloid Leukemia
  • Aggressive Systemic Mastocytosis
  • Mast Cell Leukemia (MCL)
  • Systemic Mastocytosis

Use in Cancer

Midostaurin is approved to treat:

  • Acute myeloid leukemia (AML). It is used with cytarabine and daunorubicin hydrochloride in newly diagnosed adults whose cancer has a mutation in the FLT3 gene.
  • Aggressive systemic mastocytosissystemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell leukemia in adults.

Midostaurin is also being studied in the treatment of other conditions and types of cancer.

Contraindications

  • abnormal EKG with QT changes from birth
  • pregnancy
  • a patient who is producing milk and breastfeeding
  • lung tissue problem

Strengths: 25 mg

Acute Myeloid Leukemia

  • 50 mg orally twice a day at approximately 12-hour intervals on Days 8 to 21 of each cycle of induction AND on Days 8 to 21 of each cycle of consolidation.
  • This drug is not indicated as single-agent induction therapy for acute myeloid leukemia (AML) patients.
  • Information on FDA-approved tests for the detection of FLT3 mutation in AML is available at: http://www.fda.gov/CompanionDiagnostics

Systemic Mastocytosis

  • 100 mg orally twice a day at approximately 12-hour intervals
  • Monitor for toxicity at least weekly for the first 4 weeks, every other week for the next 8 weeks, and monthly thereafter while on treatment.
  • Treatment should be continued until the disease progresses or has unacceptable toxicity.
  • For the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL)

Leukemia

  • 100 mg orally twice a day at approximately 12-hour intervals
  • Monitor for toxicity at least weekly for the first 4 weeks, every other week for the next 8 weeks, and monthly thereafter while on treatment.
  • Treatment should be continued until the disease progresses or has unacceptable toxicity.
  • For the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL)

Dose Adjustments

FOR PATIENTS WITH SYSTEMIC MASTOCYTOSIS:

ABSOLUTE COUNT (ANC) LESS THAN 1 x 10(9)/L WITHOUT MCL OR ANC LESS THAN 0.5 x 10(9)/L WITH ANC VALUE of 0.5 to 1.5 x 10(9)/L:

  • Interrupt treatment until ANC 1 x 10(9)/L or greater, then resume treatment at 50 mg twice a day; increase to 100 mg twice a day if tolerated.
  • Discontinue treatment if low ANC persists for more than 21 days and is suspected to be drug-related.

PLATELET COUNT LESS THAN 50 X 10(9)/L WITHOUT MCL OR PLATELET COUNT LESS THAN 25 x 10(9)/L WITH BASELINE PLATELET COUNT of 25 to 75 x 10(9)/L:

  • Interrupt treatment until platelet count 50 x 10(9)/L or greater, then resume treatment at 50 mg twice a day; increase to 100 mg twice a day if tolerated.
  • Discontinue treatment if low platelet count persists for more than 21 days and is suspected to be drug-related.

LESS THAN 8 g/L WITHOUT MCL OR LIFE-THREATENING WITH BASELINE HEMOGLOBIN VALUE of 8 to 10 g/L:

  • Interrupt treatment until hemoglobin 8 g/L or greater, then resume treatment at 50 mg twice a day; increase to 100 mg twice a day if tolerated.
  • Discontinue treatment if low hemoglobin persists for more than 21 days and is suspected to be drug-related.

GRADE 3/4 AND/OR DESPITE OPTIMAL ANTI-EMETIC THERAPY: Interrupt treatment for 3 days (6 doses), then resume treatment at 50 mg twice a day; increase to 100 mg twice a day if tolerated.

OTHER GRADE 3/4 NON-HEMATOLOGICAL TOXICITIES: Interrupt treatment until the event has resolved to Grade 2 or less, then resume treatment at 50 mg twice a day; increase to 100 mg twice a day if tolerated.

Side Effects

The Most Common

  • nausea
  • vomiting
  • nosebleeds
  • tiredness
  • increased sweating
  • white patches or sores on the lips or in the mouth and
  • of the hands, feet, ankles, or lower legs
  • back, bone, joint, limb, or
  • difficulty falling asleep or staying asleep
  • , , sore throat, chills, and other signs of infection
  • rapid, irregular, or pounding heartbeat
  • shortness of breath
  • chest pain
  • flushing
  • swelling of the lips, tongue, or throat
  • difficulty breathing or swallowing
  • hives
  • rash
  • itching
  • new or worsening cough
  • wheezing
  • vomiting blood or vomiting material that looks like coffee grounds
  • unusual bleeding or bruising
  • burning or pain when urinating

More common

  • Black, tarry stools
  • bloody urine
  • body aches or pain
  • bone, joint, or muscle pain
  • chest pain or tightness
  • cough
  • decreased frequency or amount of urine
  • dizziness
  • ear congestion
  • fever or chills
  • headache
  • increased thirst
  • loss of appetite
  • loss of voice
  • lower back or side pain
  • nausea
  • painful or difficult urination
  • pale skin
  • runny or stuffy nose
  • sneezing
  • sore throat
  • stomach pain
  • swelling of the face, fingers, or lower legs
  • trouble breathing
  • ulcers, sores, or white spots in the mouth
  • unusual bleeding or bruising
  • unusual tiredness or weakness
  • vomiting
  • weight gain

Rare

  • Chills
  • general feeling of discomfort or illness
  • thickening of bronchial secretions
  • Bloody nose
  • blurred vision
  • cracked lips
  • diarrhea
  • difficulty having a bowel movement (stool)
  • difficulty with moving
  • difficulty with swallowing
  • dry mouth
  • flushed, dry skin
  • fruit-like breath odor
  • headache
  • increased hunger
  • increased sweating
  • increased thirst
  • increased urination
  • muscle stiffness
  • small red or purple spots on the skin
  • sores, ulcers, or white spots on the lips, tongue, or inside the mouth
  • sweating
  • trouble sleeping
  • unexplained weight loss

Drug Interactions

Drug-Food Interactions

  • Avoid St. John’s Wort. This herb induces the CYP3A4 metabolism of midostaurin and may reduce its serum concentration.
  • Exercise caution with grapefruit products. Grapefruit inhibits the CYP3A4 metabolism of midostaurin, which may increase its serum concentration.
  • Take with food. Taking midostaurin with food increases the AUC, but it also prolongs the Tmax and reduces Cmax.

Pregnancy and Lactations

AU TGA pregnancy category: D
US FDA pregnancy category: Not Assigned

Pregnancy

Lactation

No information is available on the clinical use of midostaurin during breastfeeding. Because midostaurin and its active metabolites are 99.8% bound to plasma proteins, the amount in milk is likely to be low. The manufacturer recommends that breastfeeding be discontinued during midostaurin therapy and for at least 4 months after the last dose. Avoiding breastfeeding is particularly important when midostaurin is given with other cancer chemotherapy agents.

Why is this medication prescribed?

Midostaurin is used with other chemotherapy drugs to treat certain types of acute myeloid leukemia (AML; a type of cancer of the white blood cells). Midostaurin is also used to certain types of mastocytosis (a blood disorder in which there are too many mast cells [a certain kind of white blood cell]). Midostaurin is in a class of medications called kinase inhibitors. It works by blocking the action of the abnormal protein that signals cancer cells to multiply. This helps stop the spread of mast and cancer cells.

How should this medicine be used?

Midostaurin comes as a capsule to take by mouth. It is usually taken with food twice a day. Take midostaurin at around the same times every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take midostaurin exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Swallow the capsules whole; do not open or crush them.

If you vomit after taking midostaurin, do not take another dose. Continue your regular dosing schedule.

Your doctor may decrease your dose of midostaurin or tell you to stop taking midostaurin for a period of time or permanently during your treatment. This depends on the side effects you experience. Talk to your doctor about how you are feeling during your treatment. Continue to take midostaurin even if you feel well. Do not stop taking midostaurin without talking to your doctor.

Your doctor may tell you to take medication to prevent nausea and vomiting before each dose of midostaurin.

Ask your pharmacist or doctor for a copy of the manufacturer’s information for the patient.

Other uses for this medicine

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

What special precautions should I follow?

Before taking midostaurin,

  • tell your doctor and pharmacist if you are allergic to midostaurin, any other medications, or any of the ingredients in midostaurin capsules. Ask your pharmacist for a list of the ingredients.
  • tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, and nutritional supplements you are taking or plan to take. Be sure to mention any of the following: boceprevir (no longer available in the U.S.; Victrelis); carbamazepine (Carbatrol, Equetro, Tegretol, others); clarithromycin (Biaxin, in Prevpac); diltiazem (Cardizem, Cartia, Tiazac, others); enzalutamide (Xtandi); idelalisib (Zydelig); itraconazole (Onmel, Sporanox); ketoconazole (Nizoral); medications used to treat human immunodeficiency virus (HIV) such as cobicistat (Tybost, in Evotaz, in Genvoya, in Prezcobix, in Stribild), elvitegravir (Vitekta), indinavir (Crixivan), nelfinavir (Viracept), ritonavir (Norvir, in Kaletra, in Technivie, in Viekira), saquinavir (Invirase) and tipranavir (Aptivus); mitotane (Lysodren); nefazodone; phenytoin (Dilantin, Phenytek); posaconazole (Noxafil); rifampin (Rifadin, Rimactane, in Rifamate, in Rifater); troleandomycin (not available in the U.S.); and voriconazole (Vfend). Your doctor may need to change the doses of your medications or monitor you carefully for side effects. Many other medications may also interact with midostaurin, so be sure to tell your doctor about all the medications you are taking, even those that do not appear on this list.
  • tell your doctor what herbal products you are taking, especially St. John’s wort.
  • tell your doctor if you have or have ever had an irregular heartbeat, QT prolongation (a heart problem that may cause irregular heartbeat, fainting, or sudden death), lung, liver, or kidney disease.
  • tell your doctor if you are pregnant or plan to become pregnant. If you are a female, you should not become pregnant while you are taking midostaurin and for up to 4 months after your final dose. You will need to have a negative pregnancy test within 7 days before you start taking midostaurin. Talk to your doctor about birth control methods that will work for you. If you are male, you and your female partner should use birth control during your treatment and continue to use birth control for 4 months after your final dose. If you or your partner become pregnant while taking midostaurin, call your doctor immediately. Midostaurin may harm the fetus.
  • tell your doctor if you are breastfeeding. You should not breastfeed while you are taking midostaurin and for 4 months after your final dose.
  • you should know that this medication may decrease fertility in men and women.

  1. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/207997s000lbl.pdf
  2. https://www.fda.gov/drugs/resources-information-approved-drugs/midostaurin
  3. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/207997Orig1Orig2s000MedR.pdf
  4. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/207997Orig1Orig2s000ClinPharmR.pdf
  5. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/207997Orig1Orig2s000PharmR.pdf
  6. https://pubchem.ncbi.nlm.nih.gov/compound/Midostaurin
  7. https://pubchem.ncbi.nlm.nih.gov/compound/Midostaurin-hydrate
  8. https://www.cancer.gov/about-cancer/treatment/drugs/midostaurin
  9. https://go.drugbank.com/drugs/DB06595
  10. https://en.wikipedia.org/wiki/Midostaurin
  11. https://medlineplus.gov/druginfo/meds/a617033.html
  12. https://www.mayoclinic.org/drugs-supplements/midostaurin-oral-route/precautions/drg-20406228?p=1
  13. https://www.drugs.com/pregnancy/midostaurin.html
  14. https://www.webmd.com/drugs/2/drug-173575/midostaurin-oral/details/list-contraindications

RX Medical Knowledge Graph

Explore this medical topic

Continue through verified related conditions, investigations, medicines, and patient guides. These links are educational and do not replace professional medical advice.

RX Clinical Pathway Engine

Continue through a complete learning pathway

Move from understanding the topic to symptoms, tests, treatment, medicines, monitoring, and prevention.

Search the complete library
  1. Understand the condition Begin with the essential facts and a clear explanation of the topic.
  2. Recognize symptoms Learn common symptoms, signs, and patterns of presentation.
  3. Know when to seek help Review urgent warning signs and when professional assessment may be needed.
  4. Understand causes and risks Explore causes, risk factors, mechanisms, and contributing conditions.
  5. Explore tests and diagnosis Learn how clinicians assess the condition and which investigations may be discussed.
  6. Learn treatment approaches Review general treatment categories and management principles.
  7. Understand medicines safely Continue to medicine education, uses, precautions, and monitoring.
  8. Plan monitoring and follow-up Understand monitoring, complications, rehabilitation, and follow-up learning.
  9. Review prevention and self-care Explore prevention, healthy routines, and questions to discuss with a clinician.

Conditions & Diseases

Background, symptoms, causes, diagnosis, and care.

Explore this library

Tests & Investigations

Laboratory, imaging, screening, and diagnostic education.

Explore this library

Medicines

Uses, safety, monitoring, and related medicine knowledge.

Explore this library

Cancer Knowledge

Cancer types, screening, oncology, and treatment education.

Explore this library
Doctor visit helper

Prepare before seeing a doctor

A simple rural-patient checklist to help you explain symptoms clearly, ask better questions, and avoid unsafe self-treatment.

Safety note: This is not a prescription or diagnosis. For severe symptoms, pregnancy danger signs, children with serious illness, chest pain, breathing difficulty, stroke-like weakness, or major injury, seek urgent care.

Which doctor may help?

Start with a registered doctor or the nearest qualified health center.

What to tell the doctor

  • Write when the problem started and how it changed.
  • Bring old prescriptions, investigation reports, and current medicines.
  • Write allergies, pregnancy status, diabetes, kidney/liver disease, and major past illnesses.
  • Bring one family member if the patient is weak, elderly, confused, or a child.

Questions to ask

  • What is the most likely cause of my symptoms?
  • Which danger signs mean I should go to hospital quickly?
  • Which tests are necessary now, and which can wait?
  • How should I take medicines safely and what side effects should I watch for?
  • When should I come for follow-up?

Tests to discuss

  • Vital signs: temperature, pulse, blood pressure, oxygen saturation
  • Basic physical examination by a clinician
  • CBC, urine test, blood sugar, or imaging only when clinically needed

Avoid these mistakes

  • Do not use antibiotics, steroid tablets/injections, or strong painkillers without proper medical advice.
  • Do not hide pregnancy, kidney disease, ulcer, allergy, or blood thinner use.
  • Do not delay emergency care when danger signs are present.

Medicine safety and first-aid guide

This section is for patient education only. It does not replace a doctor, pharmacist, or emergency care.

Safe first steps

  • Avoid heavy lifting, sudden bending, and prolonged bed rest.
  • Use comfortable posture and gentle movement as tolerated.
  • Discuss physiotherapy, X-ray, or MRI only when clinically needed.

OTC medicine safety

  • For mild back pain, pain-relief medicine may be discussed with a doctor or pharmacist.
  • Avoid repeated painkiller use if you have kidney disease, stomach ulcer, uncontrolled blood pressure, or are taking blood thinners.

Avoid these mistakes

  • Do not start antibiotics without a proper medical decision.
  • Do not use steroid tablets or injections casually for quick relief.
  • Do not delay emergency care because of home remedies.

Get urgent help if

  • Back pain with leg weakness, numbness around private area, loss of urine/stool control, fever, cancer history, or major injury needs urgent care.
Medicine names, dose, and timing must be decided by a qualified clinician or pharmacist after checking age, pregnancy, allergy, other diseases, and current medicines.

For rural patients and family caregivers

Patient health record and symptom diary

Write your symptoms, medicines already taken, test results, and questions before visiting a doctor. This note stays on your device unless you print or copy it.

Doctor to discuss: Medicine doctor / pediatrician for children / qualified clinician
Tests to discuss with doctor
  • Temperature chart and hydration assessment
  • CBC with platelet count if fever persists or dengue/other infection is possible
  • Urine test, malaria/dengue tests, chest evaluation, or blood culture only when clinically indicated
Questions to ask
  • What is the most likely cause of my symptoms?
  • Which warning signs mean I should go to emergency care?
  • Which tests are really needed now?
  • Which medicines are safe for my age, pregnancy status, allergy, kidney/liver/stomach condition, and current medicines?
  • Do I need antibiotics, or is this more likely viral?

Emergency warning signs such as chest pain, severe breathing difficulty, sudden weakness, confusion, severe dehydration, major injury, or loss of bladder/bowel control need urgent medical care. Do not wait for online information.

Safe pathway to proper treatment

Care roadmap for: Midostaurin – Uses, Dosage, Side Effects, Interaction

Use this simple roadmap to understand the next safe steps. It is educational and does not replace examination by a doctor.

Go to emergency care if you notice:
  • Severe or rapidly worsening symptoms
  • Breathing difficulty, chest pain, fainting, confusion, severe weakness, major injury, or severe dehydration
Doctor / service to discuss: Qualified healthcare provider; specialist depends on symptoms and examination.
  1. Step 1

    Check danger signs first

    If danger signs are present, seek emergency care and do not wait for online information.

  2. Step 2

    Record the symptom story

    Write when symptoms started, severity, medicines already taken, allergies, pregnancy status, and test results.

  3. Step 3

    Visit a qualified clinician

    A doctor, nurse, or qualified healthcare provider can examine you and decide which tests or treatment are needed.

  4. Step 4

    Do only useful tests

    Do tests after clinical assessment. Avoid unnecessary tests, random antibiotics, or repeated medicines without diagnosis.

  5. Step 5

    Follow up and return early if worse

    If symptoms worsen, new warning signs appear, or treatment is not helping, return for review quickly.

Rural patient practical tips
  • Take a written symptom diary and all previous prescriptions/test reports.
  • Do not hide medicines already taken, even herbal or over-the-counter medicines.
  • Ask which warning signs mean urgent referral to hospital.

This roadmap is for education. A real diagnosis and treatment plan requires history, examination, and clinical judgment.

Internal learning pathway

Explore related RX articles

Related guides from RX Harun are grouped to help readers move from overview to symptoms, tests, treatment, and safe next steps.

Drugs (A - Z)
  1. Vasodilator Drugs Vasodilator drugs relax and widen blood vessels, increasing blood flow. A few examples include ACE inhibitors, ARBs, calcium…
  2. Methamphetamine Hydrochloride – Uses, Dosage, Side Effects, Interaction Methamphetamine Hydrochloride is the hydrochloride salt form of methamphetamine, an amphetamine and sympathomimetic amine with CNS stimulating properties. Methamphetamine…
  3. Methamphetamine – Uses, Dosage, Side Effects, Interactions Methamphetamine is a member of the class of amphetamines in which the amino group of (S)-amphetamine carries a methyl substituent.…
  4. Vitamin D3 – Uses, Dosage, Side Effects, Interactions DefinitionCholecalciferol is a steroid? hormone produced in the skin when exposed to ultraviolet light or obtained from…
  5. Imidazole – Uses, Dosage, Side Effects, Interactions DefinitionImidazole is an imidazole tautomer which has the migrating hydrogen at position 1. It is a conjugate base of…
  6. Carbimazole Carbimazole is a member of the class of imidazoles that is methimazole in which the nitrogen-bearing hydrogen is…