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Treatment for myelofibrosis

Read about treatment and how to cope with myelofibrosis.

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.

This helps your doctor decide on the best treatment for you.

There are 4 risk groups:

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

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 - if you have weight loss or sweats
  • your age – if you are over 65 years you are higher risk

Generally speaking, low risk myelofibrosis can be kept under control for several years in some people. In high risk myelofibrosis, survival can vary from months to a few years.

The predicted outlook of myelofibrosis depends on so many factors. So it's 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.

When red blood cells are broken down by the body they produce urea. Too much urea upsets the chemical balance in the blood and causes gout. Gout is a type of arthritis that causes pain and swelling in the joints.

Allopurinol reduces the amount of urea. You take it as a tablet.

Chemotherapy uses cell killing (cytotoxic) 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
  • chlorodeoxyadenosine

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.

To have a donor stem cell transplant you first need to have your own bone marrow cells destroyed. This means having high dose chemotherapy. You might also have total body radiotherapy. 

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. Usually this is through a central line rather than through a drip into your arm.

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 (through registers such as Anthony Nolan).

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.

This treatment is very intense and has risks. You can usually only have this if you:

  • are younger
  • are in reasonably good health, apart from your myelofibrosis
  • have a suitable donor (sibling or unrelated)

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.

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

Less common treatments

Androgens are hormones made by the adrenal glands. Doctors sometimes use man made 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 cause prostate cancer. Men will have screening for prostate cancer.

This type of treatment is not used as much these days. 

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.


How myeloproliferative disorders develop

Researchers are looking into how myeloproliferative disorders develop. Some of this research suggests that it may be better to group them depending on whether they are JAK2 positive or negative rather than on the type of myeloproliferative disorder. We need more research to find the best way to group these conditions and what the best treatment is.


Ruxolitinib is already used to treat the symptoms of myelofibrosis. Researchers want to know if this drug can be used in other ways to treat myelofibrosis. 

For example, a trial is looking at whether ruxolitinib would benefit people with earlier stage disease. These people might not have any significant symptoms and would not normally start treatment.  Researchers think that ruxolitinib could delay or lower the chances of early myelofibrosis becoming more severe in people at high risk.

You can look for trials using ruxolitinib on our clinical trials database.

Other drugs

Trials are also looking at whether ruxolitinib can be combined with other drugs to improve treatment. These other drugs include:

  • panobinostat – this blocks enzymes that cells need to grow and divide
  • BKM120 – this blocks a group of proteins called PI3K
  • azacitidine - a chemotherapy drug

Coping with myelofibrosis

Coping with a rare blood disorder can be difficult both practically and emotionally. Being well informed about your condition and possible treatments can make it easier to make decisions and cope with what happens. Your cancer specialist and Clinical Nurse Specialist are the best source of information about your situation.

There are organisations that offer help and support.

MPD Voice offers information and support to people with myeloproliferative disorders.

Leukaemia Care has information about myelofibrosis and has a message board

Last reviewed: 
06 Sep 2017
  • Ruxolitinib for treating disease-related splenomegaly or symptoms in adults with myelofibrosis. Technology appraisal guidance [TA386]
    National Institute for Health and Care Excellence (NICE). March 2016

  • Ruxolitinib
    Scottish Medicines Compendium. March, 2015

  • Classification and diagnosis of myeloproliferative neoplasms according to the 2008 World Health Organization criteria
    M. Wadleigh M1 and A.Tefferi
    International Journal of Hematology. 2010 Mar;91(2):174-9

  • Essential Haematology. 7th Edition
    AV. Hoffbrand and PAH. Moss
    Wiley Blackwell. 2016

  • JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis
    C. Harrison (and others)
    New England Journal of Medicine. 2012 Mar 1;366(9):787-98

  • Myeloproliferative neoplasms: molecular pathophysiology, essential clinical understanding, and treatment strategies
    A. Tefferi and W. Vainchenker
    Journal of Clinical Oncology. 2011 Feb 10;29(5):573-82

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