Tests and treatment for myelofibrosis

The first test to diagnose myelofibrosis is usually a blood test called a full blood count. This is to check the number of:

  • red blood cells
  • white blood cells
  • platelets

Most people with myelofibrosis have low number of red blood cells. This is called anaemia. Red blood cells contain haemoglobin, and so your haemoglobin will also be low. Haemoglobin carries oxygen around the body. So, if you are anaemic, you may feel tired or breathless.

Some people with myelofibrosis have a normal or increased level of haemoglobin in the blood.

Many people have a higher than normal white blood cell count and platelets at diagnosis. These usually drop as the condition progresses.

Other tests

Other tests you might have include:

  • an ultrasound scan of your tummy (abdomen) - to check the size of your spleen and liver
  • a bone marrow biopsy
  • an MRI scan
  • a CT scan

If these tests suggest you have myelofibrosis, you then have another blood test to look for gene Open a glossary item changes, for example in the JAK2 or CALR genes.

Doctors are learning more about genetic changes all the time. You might have tests for other gene changes.


Your treatment depends on a number of factors including:

  • your blood count at diagnosis
  • your symptoms
  • the number of immature cells you have in your blood
  • your risk group

Your risk group and outlook

There are particular factors that doctors use to work out whether your myelofibrosis is low, medium or high risk to your health. This is called the International Prognostic Scoring System (IPSS).

The Dynamic International Prognostic Scoring System (DIPSS) helps doctors work out your risk at any point during your myelofibrosis treatment.

These systems help your healthcare team decide on the best treatment for you.

There are 4 risk groups:

  • low risk
  • intermediate 1
  • intermediate 2
  • high risk

At diagnosis, your risk group depends on:

  • your white blood cell count - a high one is higher risk
  • your haemoglobin - less than 100g/L is higher risk
  • the number of blasts (immature blood cells) in your blood
  • your symptoms - for example if you have weight loss or sweats
  • your age - if you are over 65 years you are higher risk

Newer scores are sometimes available using particular DNA Open a glossary item changes inside the myelofibrosis cells. It may help to talk to your specialist about which scoring system they are using.

Generally speaking, low risk myelofibrosis can be kept under control for several years in some people. High risk myelofibrosis can be more difficult to treat.

The predicted outlook of myelofibrosis depends on so many factors. So, it is best to discuss your outlook with your own specialist. They have access to all your test results and how they have changed over time.


Your doctor can decide on the best treatment for you by looking at the scoring system and finding your risk group. They will also take into account your general health and any other medical conditions. 

You might not need to start treatment if you don’t have symptoms. Instead you have regular check ups, including blood tests to check your blood count.

Treatments might include one or more of the following:

Blood transfusions increase the number of red blood cells. This helps to control tiredness, breathlessness and weakness.

You take aspirin as a tablet. This can help to lower the number of platelets in the blood.

When red blood cells are broken down by the body, they produce uric acid. If you have too much uric acid in your blood, it can collect in the joints and cause painful swelling, called gout. Allopurinol lowers the amount of uric acid in the blood and so helps to reduce the symptoms of gout.

You have allopurinol as a tablet.

JAK2 inhibitors work by slowing or stopping the growth of cancer cells. They are also called cancer growth blockers.

JAK2 inhibitors stop the JAK2 gene from signalling to stem cells to make blood cells. An example of a JAK2 inhibitor is ruxolitinib. You take ruxolitinib as a tablet.

Ruxolitinib is used to treat an enlarged spleen and other symptoms or if you have intermediate or high risk myelofibrosis. 

Chemotherapy uses cytotoxic Open a glossary item drugs to destroy the immature cells. The drugs work by disrupting the growth of cells and stopping them from dividing. They can help to reduce enlargement of the spleen and liver.

Chemotherapy is not suitable for everyone because one of the side effects is the lowering of the number of blood cells. 

The drugs doctors use might include:

  • hydroxycarbamide
  • busulfan
  • melphalan

You have some of these drugs as tablets, or as a drip into your bloodstream (intravenous).

Pegylated interferon is a type of immunotherapy. It helps to reduce the rate at which blood cells are made.

You might have pegylated interferon if you have tried other treatments for myelofibrosis and they are not working for you. You are more likely to have this treatment as an injection just under the skin (subcutaneously). Or you might have it into the bloodstream through a drip (infusion).

Erythropoietin is a growth factor Open a glossary item which encourages your body to make more red blood cells.

Radiotherapy is the use of radiation, usually x-rays, to kill cells. Your doctor might suggest you have radiotherapy if your spleen is enlarged and you are unable to have surgery.

This helps reduce the size of your spleen, but it usually only controls symptoms for a few months.

Donor stem cell transplants are usually only suitable for people who:

  • are younger
  • are in reasonably good health, apart from your myelofibrosis
  • have a suitable donor (sibling or unrelated)
  • are in the intermediate 2 or high risk group

This is because it’s an intensive treatment and there are risks. There are several stages to a donor stem cell transplant.

Stages of a donor stem cell transplant

You have a stem cell transplant after very high doses of chemotherapy. You might have targeted drugs with the chemotherapy. You may also have radiotherapy to your whole body. This is called total body irradiation or TBI.

After this treatment you have stem cells from a donor to replace your own bone marrow cells. You have these through a drip into your bloodstream.

To be able to have intensive treatment you need a stem cell donor. The donor is someone whose stem cells match yours. The most suitable donor is usually a close relative such as a brother or sister. If you don't have a suitable family donor your doctor might look for an unrelated donor for you.

It takes from a few days to a few weeks for the donor stem cells to start making new blood cells. During this time, you will need blood and platelet transfusions. You are also at a high risk of developing infections, so you have antibiotics and anti-viral drugs to try to prevent this from happening.

Androgens are hormones made by the adrenal glands. Doctors sometimes use artificial androgens to help to control anaemia. You take them as a tablet.

There are a number of types, including danazol. Androgens have side effects including an increase in body hair and sexual desire, and retention of fluid. They can also cause changes in your liver and increase the risk of prostate cancer. Men will have screening for prostate cancer.

You might have an operation to remove your spleen (splenectomy) if it is enlarged and other treatments haven't been able to reduce the size. 

The operation has risks for someone with myelofibrosis. These include infection, bleeding and development of blood clots. So, it's not a suitable treatment for everyone.

Healthy lifestyle

Your healthcare team might discuss ways to maintain a healthy lifestyle and help with stopping smoking if you need it.

We have information on healthy diet and lifestyle on our causes of cancer and reducing your risk pages.

Support and coping

We have information on support organisations that can help you cope with the diagnosis and treatment of myelofibrosis.

Last reviewed: 
27 Jul 2020
Next review due: 
27 Jul 2023
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    JM O’Sullivan and CN Harrison

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    Electronic Medicines Compendium

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    National Institute for Health and Care Excellence, March 2016

  • Myelofibrosis

    BMJ Best Practice, Accessed June 2020

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