Myelofibrosis (MF) treatment
Currently the only treatment that can cure MF is a stem cell transplant, but other treatments aim to control the symptoms so you can continue to live the way you want.
Treatment aims and decisions
Treatment for myelofibrosis (MF) has several aims:
- to control your symptoms
- to lower your risk of blood clots or bleeding (if this is a risk for you)
- to increase the number of red blood cells you produce (if you have anaemia)
- to lower the chance of the MF developing into leukaemia
- to give you the best quality of life possible.
Your doctor will recommend a treatment based on things that are individual to you, including:
- your age and general fitness
- your blood counts (the number of blood cells of each type in your blood)
- your symptoms
- genetic tests that show which gene mutations are causing the MF
Your doctor will work out a risk score based on your test results and symptoms. This score may be low risk, intermediate-1, intermediate-2 or high risk. Some scoring systems have more levels but the point of your risk score is to help doctors plan the best treatment for you.
Your hospital team will discuss the treatment plan with you and take your wishes into account. They may suggest you take part in a clinical trial, which is an opportunity to try a new drug or combination of drugs that may be not available outside of the trial. You can read more about clinical trials.
If you have any other health conditions that could affect your heart or blood vessels (such as diabetes, high blood pressure or high cholesterol), these should be reviewed and controlled as tightly as possible. Your GP is the doctor responsible for this, while your hospital team monitors the myelofibrosis.
I’ve benefited from many different medications over the years...There are always other options if a drug stops working so well or has side effects.
- Marilyn, living with myelofibrosis since 1990
Treatment if your risk is low or intermediate-1
If your risk score is low or intermediate-1, you may not need any treatment when you are diagnosed with myelofibrosis, just regular check-ups. This is called being on active monitoring or watch and wait.
You may find it worrying or confusing to be told you have a type of blood cancer but don’t need any treatment. Read our information about active monitoring for MF and about looking after yourself. You can also connect with other people on active monitoring (watch and wait) on our online community forum.
Treatment for high platelets
If the myelofibrosis causes a high platelet count, your blood will be thicker than normal and you may be at risk of blood clots.
The main treatment for this is a low daily dose of aspirin, or an alternative called clopidogrel. This might be the only treatment you need for now.
It can feel odd to be told you will take aspirin to treat blood cancer. You may find it helpful to read our information about active monitoring (watch and wait). Though aspirin is technically a treatment, you may have feelings in common with people on watch and wait, who have regular check-ups but no cancer treatment.
Treatment if your risk is intermediate-2 or high
If you have intermediate-2 or high risk myelofibrosis (MF) you are more likely to need treatment.
There is a range of drugs that can treat MF. You may need to change to another treatment if it stops working as well, or if the side effects are severe.
Researchers are developing new drugs for MF – in particular, drugs called inhibitors. These work by blocking the signals that make cancer cells grow. So it is likely that there will be more treatment options for people with MF in the near future.
Drugs that are currently used for treating myelofibrosis are:
Ruxolitinib is a targeted drug called a JAK inhibitor. It targets something called the JAK-STAT pathway, disrupting the chemical signals that tell the myelofibrosis cells (cancer cells) to grow.
Whether you have the JAK2, CALR or MPL mutation or none of these (triple negative myelofibrosis) the drug can be effective. It helps to control the effects of a swollen spleen and other common symptoms.
Ruxolitinib is taken as tablets at home, usually twice a day. Most people who take ruxolitinib have few side effects and manage well on it.
People on ruxolitinib must avoid foods containing grapefruit or Seville oranges (including juices) as these can affect how the drug works.
Speak to your doctor if you want to have a child while taking ruxolitinib as it may harm the baby. This applies whether it is the egg or sperm that might be affected by the drug.
Fedratinib is a JAK inhibitor that targets the JAK-STAT pathway. It is recommended for people who have previously been treated with ruxolitinib, if the ruxolitinib has become less effective at managing myelofibrosis symptoms.
Fedratinib is taken as capsules at home. As with ruxolitinib you must avoid foods containing grapefruit or Seville oranges (including juices) as these can affect how the drug works.
You must not become pregnant or make someone else pregnant while taking fedratinib as it may harm the baby. Speak to your doctor first if you want to have a child.
Hydroxycarbamide is a type of drug called an anti-metabolite. It works by interfering with the DNA in cancer cells. DNA is like a set of instructions for the cell. Damaging the DNA stops cancer cells maturing, so they cannot survive for long.
Hydroxycarbamide is a drug you can take as a capsule at home. It’s classed as a type of chemotherapy, so there will be warnings on the drug packaging that may seem alarming if you’re not expecting to see them.
It is important not to become pregnant or make someone else pregnant when taking hydroxycarbamide because there is a risk to the baby. If you want to have a baby, talk to your doctor about other treatments.
Our bodies produce a substance called interferon to help us fight germs like viruses. The interferon you take to treat myelofibrosis (MF) and other diseases is a manufactured copy of natural interferon. It lowers blood counts that are too high, so is more commonly used for low risk and intermediate-1 MF.
The type of interferon used to treat MF is called pegylated interferon or PEG interferon. It’s injected under the skin – you or someone who supports you at home can be shown how to do this. Or it can be given by your GP practice nurse or district nurse if you have difficulty.
It’s important to know that some people can become depressed when taking interferon, so tell your hospital team straight away if you notice a change in your mood when you start interferon injections.
Interferon can be a good option for younger people as it doesn’t affect your fertility (your ability to have children) and is considered safe to take during pregnancy. But it is always best to check with your doctor before deciding to get pregnant.
Busulfan is an alkylating agent. This is a type of chemotherapy drug that works by interfering with the DNA in cancer cells, stopping them dividing and multiplying.
Busulfan is usually only used when other drugs have stopped working or aren’t suitable for you. It’s mainly given to older people. You must not get pregnant or make someone else pregnant when taking busulfan because it can harm the baby.
Busulfan is taken as a tablet.
More information about drugs
MPN Voice has leaflets which give more detailed information about each of these drugs used to treat myelofibrosis.
Myelofibrosis research
Many people find hope in the knowledge that scientists are researching better and kinder treatments for MF and other MPNs.
Stem cell transplants for myelofibrosis
A stem cell transplant involves having high dose chemotherapy treatment to get rid of abnormal blood stem cells, before having new stem cells put into your body, which come from somebody else (a donor). The new stem cells can then produce normal blood cells in your body. This is a way of renewing your bone marrow, which has been damaged by the myelofibrosis (MF). It is only suitable for a small number of people with MF.
A transplant is an intensive treatment that can have serious complications. It involves having chemotherapy to destroy the abnormal cells before having the donor’s cells infused through a vein into your blood. A stem cell transplant can provide a cure for MF, but you need to be the right age and fitness level to have this intensive treatment. Doctors must be sure that the benefits outweigh the risks.
You need to get new stem cells from a donor who is a good match. If they are fit and well, brothers and sisters (siblings) are often the first people to be tested to see if they are a match. This might be done just to see if a stem cell transplant is a future option, even if you don’t need one immediately.
With siblings, there’s a 1 in 4 (25%) chance of them being a perfect match, and a 1 in 2 (50%) chance of them being a half-match to your tissue.
If a full match can’t be found in your family, or you don’t have any suitable relatives, then your hospital team will look for an unrelated matched donor on UK and international stem cell registers. Donors are all volunteers. Stem cells from a half-matched sibling may be an option if it’s difficult to find an unrelated matched donor.
Our free booklet, Blood stem cell and bone marrow transplants: The seven steps explains what happens before, during and after a stem cell transplant.
The African Caribbean Leukaemia Trust (ACLT) can support you to find a donor if you are from a Black, mixed heritage or other minoritised ethnic background.
Clinical trials for myelofibrosis
Clinical trials are research projects that test new drugs or combinations of drugs on people. All the drugs will have undergone thorough safety checks before being included in a trial. Trials look for better and kinder treatments and are a way of accessing a treatment that may not be generally available. They also provide vital information that will help doctors treat people effectively in the future.
Many people with myelofibrosis and other types of MPN have opportunities to take part in clinical trials, but it’s your choice whether or not to take part. We have a clinical trials service to talk through the process and help you decide what’s best for you. If you are interested, they can also look for trials that might be relevant for you to take part in.
Read more about research and clinical trials for people with MPNs and other blood cancers. We are able to fund these projects thanks to the generous supporters who give and raise money to beat blood cancer.
Treatments for anaemia (low red blood cells)
Many people with myelofibrosis have anaemia, a lack of red blood cells. This can cause breathlessness, dizziness and tiredness. There is a range of treatments doctors may recommend to help with anaemia:
- Erythropoietin (EPO) is a type of drug called a growth factor which stimulates your bone marrow to make more red blood cells. It is given as an injection under the skin.
- Danazol is a mild steroid which can help control anaemia. It is taken as a tablet.
- Blood transfusions can boost your red blood cells and improve the symptoms of anaemia.
- A new JAK inhibitor called momelotinib helps with anaemia and other symptoms, including shrinking a swollen spleen.
Managing your infection risk
Because myelofibrosis (MF) can affect your white blood cells, people with MF tend to have weaker immune systems than normal. This makes it harder to fight off infections such as colds, flu, covid-19 and pneumonia, so it’s important to get all the recommended vaccinations.
Your hospital team may offer you antibiotics to prevent bacterial infections. This is particularly likely if you are taking ruxolitinib, which sometimes causes old infections to come back again.
If you think you have an infection, tell your hospital team straight away. Infections need to be treated quickly or they can become serious. See our information about infection risk and blood cancer, including the symptoms of an infection, which aren’t always as obvious as you might think.
Treatment for the spleen
Treatments for myelofibrosis will usually help if you have a swollen spleen. However, if your spleen is very swollen and painful, you may need direct treatment to manage the symptoms.
In a few cases, doctors recommend having an operation to remove your spleen (a splenectomy). You can live without a spleen, but there are risks involved and it is very rare to have this operation.
Sometimes radiotherapy to the spleen can relieve symptoms on a temporary basis.
Monitoring your treatment
You will have regular check-ups throughout your treatment for myelofibrosis. You will usually have a full blood count (FBC) before each appointment, and your doctor or nurse will discuss the results with you and adjust your treatment if needed. They may feel (palpate) your spleen to see if it is swollen.
They will also ask if there have been any changes in your symptoms, or any new symptoms. So it’s a good idea to keep a note of any changes in how you feel, physically and emotionally.
Occasionally, if your doctor needs more information, you may have other tests, such as a blood film, ultrasound scan or bone marrow biopsy, but these are not routine. See our page about tests for more information.
Talk to other people affected by blood cancer
Hear from and connect with people who understand.