Rydapt: Uses, Dosage & Side Effects
A multi-targeted protein kinase inhibitor used to treat FLT3-mutation-positive acute myeloid leukemia and advanced systemic mastocytosis
Rydapt (midostaurin) is a multi-targeted protein kinase inhibitor approved for two distinct indications in adults. It is used in combination with standard chemotherapy for the treatment of newly diagnosed FLT3-mutation-positive acute myeloid leukemia (AML), and as monotherapy for aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL). Midostaurin works by blocking the activity of abnormal protein kinases, including mutant FLT3 and KIT D816V, that drive the growth and survival of malignant cells. Rydapt is taken orally as soft capsules twice daily with food and requires a prescription from a specialist physician.
Quick Facts: Rydapt
Key Takeaways
- Rydapt (midostaurin) is a protein kinase inhibitor that targets FLT3 mutations in AML and KIT D816V mutations in advanced systemic mastocytosis, blocking the signaling pathways that drive malignant cell proliferation and survival.
- For AML, Rydapt is always used in combination with standard induction and consolidation chemotherapy, followed by up to 12 months of single-agent maintenance therapy; it is not used as a standalone treatment for leukemia.
- Strong CYP3A4 inducers such as rifampicin, carbamazepine, phenytoin, enzalutamide, and St. John's wort are strictly contraindicated during Rydapt therapy as they can dramatically reduce midostaurin blood levels.
- Rydapt capsules must always be taken with food and swallowed whole; the capsules contain ethanol (alcohol) and macrogolglycerol hydroxystearate, which may be relevant for patients with certain conditions.
- Women of childbearing potential must use effective contraception during treatment and for at least 4 months after the last dose; Rydapt may impair fertility in both men and women.
What Is Rydapt and What Is It Used For?
Rydapt contains the active substance midostaurin, which belongs to a class of medicines known as protein kinase inhibitors. These drugs work by interfering with the activity of specific enzymes (kinases) that play a critical role in the signaling pathways that control cell growth, division, and survival. When these kinases become abnormally active due to genetic mutations, they can drive the uncontrolled growth of certain types of blood cells, leading to cancer.
Midostaurin is a multi-targeted kinase inhibitor, meaning it blocks several different kinases simultaneously. Its primary targets include FLT3 (fms-like tyrosine kinase 3) in both wild-type and mutant forms (including FLT3-ITD and FLT3-TKD mutations), KIT (including the D816V mutation), PDGFR-alpha and -beta, VEGFR-2, and members of the protein kinase C (PKC) family. By inhibiting these enzymes, midostaurin disrupts the signals that tell cancer cells to keep growing and dividing, ultimately leading to cell cycle arrest and programmed cell death (apoptosis).
Acute Myeloid Leukemia (AML)
Rydapt is approved for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults whose leukemia cells carry a defect (mutation) in the FLT3 gene. AML is a type of blood cancer that originates in the bone marrow and affects a group of white blood cells called myeloid cells. In AML, the bone marrow produces large numbers of abnormal, immature white blood cells (blasts) that cannot function properly and crowd out healthy blood cells. Approximately 25-30% of AML patients harbor FLT3 mutations, which are associated with a more aggressive disease course and poorer prognosis compared to FLT3-wild-type AML.
In the treatment of AML, Rydapt is always used in combination with standard chemotherapy regimens, including induction therapy (typically daunorubicin and cytarabine) and consolidation therapy (high-dose cytarabine). After completing chemotherapy, patients may continue receiving Rydapt as single-agent maintenance therapy for up to 12 months. The landmark RATIFY clinical trial demonstrated that adding midostaurin to standard chemotherapy significantly improved overall survival in patients with FLT3-mutated AML.
Advanced Systemic Mastocytosis
Rydapt is also approved for the treatment of advanced systemic mastocytosis (advSM) in adults, encompassing three subtypes: aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). These are rare conditions characterized by the accumulation of too many mast cells (a type of white blood cell involved in allergic and inflammatory responses) in various organs, including the bone marrow, liver, spleen, and gastrointestinal tract.
In advanced systemic mastocytosis, the excessive mast cells release large quantities of chemical mediators, including histamine, causing symptoms such as severe flushing, abdominal pain, diarrhea, and potentially life-threatening anaphylaxis-like episodes. The KIT D816V mutation is found in approximately 80-95% of patients with systemic mastocytosis and is a key driver of the disease. Midostaurin effectively inhibits the KIT D816V mutant kinase, reducing the proliferation and survival of malignant mast cells and leading to improvements in organ function, symptom burden, and quality of life.
What Should You Know Before Taking Rydapt?
Contraindications
You must not take Rydapt if you are allergic to midostaurin or any of the other ingredients in the capsules. Additionally, Rydapt must not be used concurrently with certain medications that are strong inducers of the CYP3A4 enzyme, as these can dramatically reduce midostaurin blood levels and render the treatment ineffective. The contraindicated medications include:
- Rifampicin – used to treat tuberculosis
- Carbamazepine or phenytoin – used to treat epilepsy
- Enzalutamide – used to treat prostate cancer
- St. John's wort (Hypericum perforatum) – an herbal remedy for depression
If you are currently taking any of these medications and need to start Rydapt, your doctor will need to find suitable alternatives before initiating treatment. Conversely, if you are already on Rydapt and a new prescription for one of these drugs becomes necessary, inform your doctor immediately so that an alternative can be arranged.
Warnings and Precautions
Before starting treatment with Rydapt, it is important to discuss your full medical history with your healthcare team. Particular attention should be given to the following conditions, as they may require additional monitoring or adjustments to your treatment plan:
- Infections: Active infections should be treated before starting Rydapt, as the medication can suppress certain components of the immune system. During treatment, report any new signs of infection (fever, sore throat, mouth ulcers) promptly, as they may indicate low white blood cell counts (neutropenia).
- Heart disease: Rydapt can affect the heart's electrical activity and may cause QT prolongation visible on an electrocardiogram (ECG). Patients with pre-existing cardiac conditions should be monitored closely, and regular ECGs may be recommended.
- Lung problems: Non-infectious interstitial lung disease and pneumonitis have been reported during Rydapt treatment. New or worsening respiratory symptoms such as cough, chest pain, difficulty breathing, or shortness of breath should be reported immediately.
- Kidney disease: Impaired kidney function may affect how your body processes midostaurin and its metabolites. Your doctor may adjust the monitoring schedule accordingly.
- Fever, sore throat, or mouth ulcers (may indicate very low white blood cell counts)
- New or worsening cough, chest pain, difficulty breathing, or shortness of breath (may indicate lung problems)
- Chest pain or discomfort, feeling faint, dizziness, blue discoloration of lips or extremities, swelling of legs (may indicate heart problems)
Monitoring During Treatment
Your doctor will perform regular blood tests throughout your treatment with Rydapt to monitor several important parameters. These include complete blood counts (white blood cells, red blood cells, and platelets) and electrolyte levels (calcium, potassium, and magnesium). Heart function will be assessed periodically through electrocardiograms, and lung function will be monitored clinically. These checks are essential for detecting potential complications early and allowing timely dose adjustments or supportive care.
Children and Adolescents
Rydapt is not approved for use in children and adolescents under 18 years of age. When used in combination with chemotherapy, Rydapt may cause a severe reduction in certain types of blood cells in pediatric patients, and there is insufficient data to establish safe dosing guidelines for this age group.
Pregnancy and Breastfeeding
Rydapt may cause harm to an unborn baby and is not recommended during pregnancy. If you are pregnant, think you may be pregnant, or are planning to become pregnant, you should consult your doctor before taking this medicine. Your doctor will perform a pregnancy test before treatment begins to confirm you are not pregnant.
Women of childbearing potential must use an effective method of contraception while taking Rydapt and for at least 4 months after the last dose. You should discuss the most appropriate contraceptive method with your doctor. If you become pregnant or suspect pregnancy during treatment, inform your doctor immediately.
Breastfeeding must be discontinued during treatment with Rydapt and for at least 4 months after the final dose, as it is unknown whether midostaurin or its metabolites pass into breast milk. Rydapt may also impair fertility in both men and women, and this should be discussed with your healthcare provider before starting treatment.
Driving and Operating Machinery
Exercise particular caution when driving or using machinery while taking Rydapt, as dizziness and vertigo are known side effects of the medication. If you experience these symptoms, avoid activities that require alertness until you feel well enough to do so safely.
Important Information About Excipients
Rydapt soft capsules contain anhydrous ethanol (alcohol). The maximum daily dose of 200 mg contains approximately 666 mg of alcohol, equivalent to about 14% by volume of anhydrous ethanol. This is roughly equivalent to 17 ml of beer or 7 ml of wine. While this amount is generally too small to cause noticeable effects, it may be a concern for patients with alcohol dependence, epilepsy, liver disease, or those who are pregnant or breastfeeding.
Rydapt also contains macrogolglycerol hydroxystearate (castor oil derivative), which may cause stomach discomfort and diarrhea in some patients.
How Does Rydapt Interact with Other Drugs?
Midostaurin is primarily metabolized in the liver by the cytochrome P450 enzyme CYP3A4. This means that other drugs which strongly affect CYP3A4 activity can significantly alter midostaurin blood levels, either reducing its effectiveness or increasing the risk of side effects. It is essential to inform your doctor or pharmacist about all medications you are currently taking, have recently taken, or plan to take, including prescription drugs, over-the-counter medicines, vitamins, and herbal supplements.
Major Interactions (Contraindicated)
The following medications must be avoided during Rydapt treatment, as they are strong CYP3A4 inducers that can dramatically lower midostaurin concentrations in the blood, potentially making the treatment ineffective:
| Drug | Therapeutic Use | Interaction Type | Clinical Significance |
|---|---|---|---|
| Rifampicin | Tuberculosis treatment | Strong CYP3A4 inducer | Dramatically reduces midostaurin levels; contraindicated |
| Carbamazepine | Epilepsy treatment | Strong CYP3A4 inducer | Dramatically reduces midostaurin levels; contraindicated |
| Phenytoin | Epilepsy treatment | Strong CYP3A4 inducer | Dramatically reduces midostaurin levels; contraindicated |
| Enzalutamide | Prostate cancer treatment | Strong CYP3A4 inducer | Dramatically reduces midostaurin levels; contraindicated |
| St. John's wort | Herbal antidepressant | Strong CYP3A4 inducer | Dramatically reduces midostaurin levels; contraindicated |
Moderate Interactions (Use with Caution)
Several other medications may interact with Rydapt and may require dose adjustments, closer monitoring, or substitution with alternative therapies. Inform your doctor if you are taking any of the following:
| Drug | Therapeutic Use | Interaction Type | Clinical Action |
|---|---|---|---|
| Ketoconazole | Antifungal | Strong CYP3A4 inhibitor | May increase midostaurin levels; monitor for toxicity |
| Clarithromycin | Antibiotic | Strong CYP3A4 inhibitor | May increase midostaurin levels; consider alternatives |
| Ritonavir | HIV treatment | Strong CYP3A4 inhibitor | May increase midostaurin levels; use with caution |
| Efavirenz | HIV treatment | Moderate CYP3A4 inducer | May reduce midostaurin levels; monitor efficacy |
| Nefazodone | Antidepressant | Strong CYP3A4 inhibitor | May increase midostaurin levels; use alternatives |
| Bupropion | Antidepressant | CYP2B6 interaction | Monitor for altered drug levels; discuss with doctor |
| Atorvastatin / Rosuvastatin | Cholesterol management | CYP3A4/OATP substrate | May have altered levels; monitor for myopathy |
| Tizanidine | Muscle relaxant | CYP1A2 substrate | Midostaurin may increase levels; monitor for sedation |
| Chlorzoxazone | Muscle spasm treatment | CYP2E1 substrate | Monitor for altered drug effects |
Always inform your doctor before starting any new medication while on Rydapt treatment, including over-the-counter drugs, supplements, and herbal products. Your doctor can assess potential interactions and make appropriate adjustments to your treatment plan.
What Is the Correct Dosage of Rydapt?
Always take Rydapt exactly as prescribed by your doctor or pharmacist. Do not exceed the dose recommended by your physician. The dosage depends on the condition being treated and your individual response to the medication.
Adults with AML
AML Dosage Regimen
The standard dose is 50 mg (2 capsules of 25 mg) taken twice daily, approximately 12 hours apart (e.g., with breakfast and with dinner). In AML, Rydapt is always given together with chemotherapy drugs during the induction and consolidation phases. After completing chemotherapy, you will continue taking Rydapt alone as maintenance therapy for up to 12 months. Your doctor will determine the exact duration based on your response.
Adults with Advanced Systemic Mastocytosis (ASM, SM-AHN, or MCL)
Mastocytosis Dosage Regimen
The standard dose is 100 mg (4 capsules of 25 mg) taken twice daily, approximately 12 hours apart. In advanced systemic mastocytosis, Rydapt is used as a long-term monotherapy, potentially for months or years. Your doctor will regularly assess your condition to determine whether continued treatment is appropriate and may adjust the dose or temporarily interrupt treatment based on your response and any side effects.
How to Take Rydapt
- Take with food: Always take Rydapt with a meal to improve absorption and reduce gastrointestinal side effects.
- Consistent timing: Take your doses at approximately the same times each day, about 12 hours apart (e.g., with breakfast and with your evening meal).
- Swallow whole: Swallow the capsules whole with a glass of water. Do not open, crush, or chew the capsules, as this may alter the dosing and cause an unpleasant taste.
- If you vomit: If you vomit after swallowing the capsules, do not take additional capsules. Wait until your next scheduled dose.
Children
Rydapt is not approved for use in patients under 18 years of age, particularly in combination with chemotherapy, due to the risk of severe blood cell count reductions.
Missed Dose
If you forget to take a dose of Rydapt, do not take the missed dose. Simply take your next dose at the regularly scheduled time. Do not take a double dose to make up for a missed one. Wait until your next planned dosing time and continue with your normal schedule.
Overdose
If you take more capsules than prescribed, or if someone else takes your medication, contact your doctor or go to the nearest emergency department immediately. Bring the medication packaging with you, as medical treatment may be necessary. Symptoms of overdose have not been well characterized in clinical trials, but may include intensification of known side effects such as nausea, vomiting, and gastrointestinal symptoms.
Stopping Rydapt without consulting your doctor may cause your condition to worsen. Continue taking the medication for as long as your doctor tells you to, even if you feel well. Your doctor will schedule regular check-ups to assess whether the treatment is working effectively.
What Are the Side Effects of Rydapt?
Like all medicines, Rydapt can cause side effects, although not everybody gets them. The types and frequency of side effects vary between the two main indications (AML and advanced systemic mastocytosis) due to differences in the disease itself, the concomitant medications used, and the dosage. Most side effects are mild to moderate and generally resolve after a few weeks of treatment. However, some side effects can be serious and require immediate medical attention.
- Difficulty breathing or swallowing
- Dizziness
- Swelling of the face, lips, tongue, or throat
- Severe skin itching with red rash or raised bumps
Side Effects in AML Patients
AML patients receive Rydapt in combination with intensive chemotherapy, so many of the observed side effects reflect the combined toxicity of the treatment regimen. The following side effects have been reported:
Very Common (more than 1 in 10 patients)
Affects >10% of patients
- Catheter site infections
- Petechiae (small red or purple spots on the skin)
- Insomnia (difficulty sleeping)
- Headache
- Shortness of breath (dyspnea)
- Abnormal ECG results (QT prolongation)
- Dizziness or lightheadedness (low blood pressure)
- Nosebleeds
- Sore throat
- Mouth ulcers (stomatitis)
- Nausea and vomiting
- Upper abdominal pain
- Hemorrhoids
- Excessive sweating
- Skin rash with peeling or scaling (exfoliative dermatitis)
- Back pain, joint pain (arthralgia)
- Fever
- High blood sugar (hyperglycemia)
- Electrolyte disturbances (high sodium, low potassium)
- Bruising and bleeding (abnormal coagulation)
- Elevated liver enzymes (ALT, AST)
Common (up to 1 in 10 patients)
Affects 1–10% of patients
- Upper respiratory tract infections
- High calcium levels (hypercalcemia)
- Fainting (syncope)
- Involuntary trembling
- High blood pressure
- Rapid heart rate (sinus tachycardia)
- Fluid around the heart (pericardial effusion)
- Fluid in the lungs (pleural effusion)
- Swollen eyelids
- Rectal and anal discomfort
- Abdominal pain, constipation
- Dry skin
- Eye pain, blurred vision (keratitis)
- Neck pain, bone pain, limb pain
- Weight gain
- Blood clot in catheter
- High uric acid levels
Not Known (frequency cannot be estimated)
Reported from post-marketing surveillance
- Raised, painful, red to dark reddish-purple skin patches or sores mainly on arms, legs, face, and neck, with fever (acute febrile neutrophilic dermatosis / Sweet syndrome)
Side Effects in Mastocytosis Patients (ASM, SM-AHN, MCL)
Patients with advanced systemic mastocytosis receive Rydapt as monotherapy at a higher dose (100 mg twice daily), and the side effect profile differs from that seen in AML patients on combination therapy. The following side effects have been reported:
Very Common (more than 1 in 10 patients)
Affects >10% of patients
- Urinary tract infections
- Upper respiratory tract infections
- Headache and dizziness
- Shortness of breath (dyspnea)
- Cough
- Fluid in the lungs (pleural effusion)
- Abnormal ECG results (QT prolongation)
- Nosebleeds
- Nausea, vomiting, diarrhea, constipation
- Swollen limbs (peripheral edema)
- Severe fatigue
- Fever
- High blood sugar (hyperglycemia)
- Jaundice (yellowing of skin and eyes)
- Elevated pancreatic enzymes (lipase, amylase) and liver enzymes (ALT, AST)
Common (up to 1 in 10 patients)
Affects 1–10% of patients
- Involuntary trembling
- Bronchitis, oral herpes, cystitis
- Sinusitis, erysipelas (skin infection)
- Shingles (herpes zoster)
- Attention difficulties
- Vertigo (spinning sensation)
- Bruising (hematoma)
- Stomach upset, indigestion
- General weakness (asthenia)
- Chills, generalized swelling
- Weight gain
- Contusions, falls
- Low blood pressure, sore throat
- Rapid weight gain
Serious Side Effects (Both Indications)
Regardless of the indication, certain serious side effects require immediate medical attention. Contact your healthcare team immediately if you experience any of the following:
- Low blood cell counts: Weakness, unexplained bleeding or bruising, frequent infections with fever, chills, sore throat, or mouth ulcers
- Pulmonary toxicity: Fever, cough with or without sputum, chest pain, difficulty breathing, or wheezing (may indicate interstitial lung disease, pneumonitis, or acute respiratory distress syndrome)
- Sepsis: Infections with fever, low blood pressure, decreased urine output, rapid pulse, and rapid breathing
- Gastrointestinal bleeding: Vomiting blood or black/bloody stools (particularly in mastocytosis patients)
How Should You Store Rydapt?
Proper storage of Rydapt is important to maintain the medication's effectiveness and safety throughout its shelf life. The following storage guidelines should be observed:
- Temperature: No special temperature requirements. Store at room temperature.
- Packaging: Keep the capsules in the original blister packaging to protect them from moisture.
- Expiry date: Do not use Rydapt after the expiry date printed on the carton and blister pack (after "EXP"). The expiry date refers to the last day of the stated month.
- Damaged packaging: Do not use the medicine if the packaging appears damaged or shows signs of tampering.
- Children: Keep out of the sight and reach of children at all times.
- Disposal: Do not dispose of medicines via wastewater or household waste. Ask your pharmacist how to properly dispose of medicines no longer in use. These measures help protect the environment.
Rydapt 25 mg soft capsules are pale orange, oblong capsules printed with "PKC NVR" in red. They are available in blister packs containing 56 capsules (2 packs of 28) or 112 capsules (4 packs of 28). Not all pack sizes may be marketed in your country.
What Does Rydapt Contain?
Understanding the full composition of Rydapt is important, particularly for patients with known allergies or sensitivities to specific excipients. Each Rydapt soft capsule contains:
Active Ingredient
Midostaurin 25 mg – the protein kinase inhibitor responsible for the therapeutic effect.
Inactive Ingredients (Excipients)
- Macrogolglycerol hydroxystearate (castor oil derivative) – may cause stomach discomfort and diarrhea
- Gelatin – capsule shell component
- Macrogol (polyethylene glycol)
- Glycerol
- Anhydrous ethanol (alcohol) – see warnings section regarding alcohol content
- Corn oil mono-di-triglycerides
- Titanium dioxide (E171) – coloring agent
- All-rac-alpha-tocopherol (vitamin E)
- Yellow iron oxide (E172) – coloring agent
- Red iron oxide (E172) – coloring agent
- Carmine (E120) – coloring agent for the printing ink
- Hypromellose – printing ink component
- Propylene glycol – printing ink component
- Purified water
The marketing authorization holder is Novartis Europharm Limited, Dublin, Ireland. Rydapt is manufactured by Novartis Farmacéutica S.A. in Barcelona, Spain, and Novartis Pharma GmbH in Nuremberg, Germany.
Frequently Asked Questions About Rydapt
Rydapt (midostaurin) is used to treat two main conditions in adults. First, it is approved for newly diagnosed FLT3-mutation-positive acute myeloid leukemia (AML) in combination with standard chemotherapy. Second, it is used as monotherapy for advanced systemic mastocytosis, including aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), and mast cell leukemia (MCL). It works by blocking protein kinases that drive the growth and survival of malignant cells.
Rydapt should be taken orally twice daily, approximately 12 hours apart, always with food. The capsules must be swallowed whole with water and should never be opened, crushed, or chewed. For AML, the dose is 50 mg (2 capsules) twice daily. For advanced systemic mastocytosis, the dose is 100 mg (4 capsules) twice daily. If you vomit after taking the capsules, do not take extra capsules; wait until your next scheduled dose.
No, Rydapt is not recommended during pregnancy as it may harm the developing fetus. Women of childbearing potential must use effective contraception during treatment and for at least 4 months after the last dose. A pregnancy test is required before starting treatment. Breastfeeding must also be avoided during treatment and for at least 4 months after the final dose. Rydapt may impair fertility in both men and women, and patients should discuss fertility preservation options with their doctor before starting treatment.
Yes, Rydapt soft capsules contain anhydrous ethanol (alcohol). The maximum daily dose of 200 mg contains approximately 666 mg of alcohol, equivalent to about 14% by volume. This amounts to roughly 17 ml of beer or 7 ml of wine. While this small amount generally does not produce noticeable effects, it may be relevant for patients with alcohol dependence, epilepsy, liver disease, or those who are pregnant or breastfeeding. Discuss any concerns with your doctor.
Strong CYP3A4 inducers must be completely avoided during Rydapt treatment, as they dramatically reduce midostaurin blood levels and can render the treatment ineffective. These include rifampicin (tuberculosis), carbamazepine and phenytoin (epilepsy), enzalutamide (prostate cancer), and St. John's wort (herbal antidepressant). Additionally, strong CYP3A4 inhibitors like ketoconazole, clarithromycin, and ritonavir may increase midostaurin levels and require monitoring or dose adjustments. Always inform your doctor about all medications, supplements, and herbal products you take.
The treatment duration depends on the indication. For AML, Rydapt is first used during chemotherapy induction and consolidation, then continued as single-agent maintenance therapy for up to 12 months after chemotherapy completion. For advanced systemic mastocytosis, Rydapt is a long-term treatment that may continue for months or years, as long as the medication remains effective and side effects are manageable. Your doctor will regularly assess your response and determine the appropriate treatment duration.
References
- European Medicines Agency (EMA). Rydapt (midostaurin) – Summary of Product Characteristics. Last updated 2025. Available from: EMA – Rydapt EPAR.
- U.S. Food and Drug Administration (FDA). Rydapt (midostaurin) Prescribing Information. Novartis Pharmaceuticals Corporation. Revised 2024.
- Stone RM, Mandrekar SJ, Sanford BL, et al. Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation. N Engl J Med. 2017;377(5):454-464. doi:10.1056/NEJMoa1614359.
- Gotlib J, Kluin-Nelemans HC, George TI, et al. Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis. N Engl J Med. 2016;374(26):2530-2541. doi:10.1056/NEJMoa1513098.
- National Comprehensive Cancer Network (NCCN). Clinical Practice Guidelines in Oncology: Acute Myeloid Leukemia. Version 1.2025.
- Dohner H, Wei AH, Appelbaum FR, et al. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN. Blood. 2022;140(12):1345-1377. doi:10.1182/blood.2022016867.
- World Health Organization (WHO). WHO Model List of Essential Medicines – 23rd List, 2023. Geneva: WHO; 2023.
- Valent P, Akin C, Metcalfe DD. Mastocytosis: 2016 updated WHO classification and novel emerging treatment concepts. Blood. 2017;129(11):1420-1427. doi:10.1182/blood-2016-09-731893.
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