midostaurin at a glance
Medicine class
Protein kinase inhibitor targeting several kinases, including FLT3 and KIT.
AML testing
A validated test must confirm an FLT3 mutation before treatment for acute myeloid leukaemia.
Administration
Take with food and swallow the soft capsules whole with water.
Specialist treatment
Treatment is started by a clinician experienced in anticancer therapy.
What is midostaurin used for?
UK-licensed uses in adults are:
Newly diagnosed FLT3 mutation-positive acute myeloid leukaemia, used with specified induction and consolidation chemotherapy and then, in patients with a complete response, as single-agent maintenance treatment.
Aggressive systemic mastocytosis.
Systemic mastocytosis with an associated haematological neoplasm.
Mast cell leukaemia.
How does midostaurin work?
Midostaurin blocks several signalling enzymes called kinases. In FLT3-mutated leukaemia it suppresses abnormal FLT3 signalling, helping stop malignant cells dividing and promoting cell death. It also inhibits abnormal KIT signalling involved in mast-cell growth, survival and mediator release.
midostaurin preparations and strengths
Capsule
Route: Oral
Strengths: 25 mg
How to take midostaurin
Follow the specialist treatment plan exactly.
Take the capsules with food.
Swallow them whole with a glass of water.
Do not open, crush or chew the capsules.
If you vomit after taking the capsules, do not take replacement capsules; wait until the next scheduled administration.
midostaurin side effects
Common or expected effects
- Nausea or vomiting
- Diarrhoea, constipation or abdominal discomfort
- Headache
- Fever or fatigue
- Mouth soreness
- Skin rash, dryness or peeling
- Back, joint or limb pain
- Swelling or weight increase
- Changes in blood counts, electrolytes, blood glucose or liver tests
Get urgent medical advice
- Fever or other evidence of serious infection, particularly with reduced white blood cells
- New or worsening cough, chest pain or breathlessness, which may indicate pneumonitis or interstitial lung disease
- Severe breathing difficulty or unusually rapid breathing
- Fainting, chest discomfort, marked swelling or other possible heart problems
- Unusual bleeding, black stools or vomiting blood
- Difficulty breathing or swallowing, facial or throat swelling, or a severe itchy raised rash
midostaurin in pregnancy
Midostaurin may harm a developing baby and is not recommended during pregnancy. A pregnancy test is advised before treatment, with effective contraception during treatment and for at least four months after it stops. Anyone who becomes pregnant or is planning pregnancy should contact their specialist promptly.
midostaurin while breastfeeding
It is unknown whether midostaurin enters human milk, but serious effects in a breastfed baby are possible. Breastfeeding should stop during treatment and for at least four months afterwards.
midostaurin interactions
Provide the specialist team with a complete list of prescribed, non-prescription and herbal products before starting anything new.
Rifampicin, carbamazepine, phenytoin, enzalutamide and St John’s wort
These potent CYP3A4 inducers substantially reduce midostaurin exposure and must not be used concurrently.
Strong CYP3A4 inhibitors, including ketoconazole, itraconazole, clarithromycin and ritonavir
These can increase midostaurin exposure; alternatives should be considered or toxicity monitored closely.
Medicines that prolong the QT interval
They may add to the risk of abnormal cardiac repolarisation, so ECG assessment may be needed.
Common questions about midostaurin
Answers are fully visible for fast scanning and source review.
Do I need an FLT3 test before midostaurin?
Yes, when it is being considered for AML. A validated test must confirm an FLT3-ITD or FLT3-TKD mutation.
Can midostaurin be used without chemotherapy?
Yes, in its licensed advanced systemic mastocytosis indications. It may also be used alone in the maintenance phase for eligible AML patients who have achieved a complete response.
Should midostaurin be taken with food?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.