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Treatment for myelodysplasia

Read about the different treatments for myelodysplastic syndrome (MDS).

The type of treatment you have depends on:

  • your type of MDS
  • your risk group
  • whether you have any other health conditions

The aim of treatment is to get the number and type of blood cells in the bloodstream back to normal.

For MDS that is classed as low risk, you are likely to have very few or no symptoms. You might not need treatment at first. Instead you will have regular check ups including blood tests. At some point, most people need to have treatment to control the symptoms of a low blood cell count. This is called supportive treatment.

For intermediate 2 or high risk MDS you may need prompt treatment. Treatments include chemotherapy or a donor stem cell transplant.

The only way to cure MDS is to have intensive treatment with a stem cell transplant from a donor. But unfortunately this type of treatment is not suitable for everyone.

Supportive treatment

Supportive treatment aims to help control the symptoms of MDS. The supportive treatment you need will depend on which type of MDS you have. You might need a combination of treatments.


You may have transfusions to treat your MDS. If your red blood cells are low, you have a drip containing red blood cells. If your platelets are low you have a drip of a clear fluid containing platelets.

Having a lot of blood transfusions can cause a build up of iron in your body. Red blood cells contain iron and your body stores this. But too much iron in your body can damage your heart and liver. To stop this, you might need to take drugs to get rid of the excess iron.

Growth factors

Growth factors are drugs that encourage your bone marrow to make more blood cells. We know from research that this can reduce the number of blood transfusions you need.

Erythropoetin is a growth factor that increases the number of red blood cells. Granulocyte colony stimulating factor (G-CSF) is a growth factor that increases the number of white blood cells. Which one you have depends on your symptoms and blood counts. You have both of these drugs as injections just under the skin (subcutaneously).


You might also need to take antibiotics to treat infections.

Immunosuppression treatment

Sometimes with MDS, white blood cells called T lymphocytes affect normal blood cell production. Immunosuppressant drugs work by making T lymphocytes less active.

Immunosuppressant drugs called anti thymocyte globulin (ATG) and cyclosporin can sometimes be used to treat a certain type of MDS but this treatment is not suitable for everyone. 


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.

Which chemotherapy you have depends on your type of MDS and your risk group. You might have chemotherapy as a tablet or as an injection into a vein. You could have just one drug or a combination of drugs.

For intermediate 2 or high risk MDS, you are likely to have chemotherapy similar to that used for acute myeloid leukaemia (AML). These include:

  • cytarabine
  • fludarabine (Fludara)
  • idarubicin (Zavedos)
  • azacitidine (Vidaza)
  • clofarabine (Evoltra)

The National Institute for Health and Care Excellence (NICE) have recommended that azacitidine should be available for people who have intermediate risk 2 or high risk MDS, and are not able to have high dose treatment with a stem cell transplant.

Intensive treatment with a stem cell transplant

To have a donor stem cell transplant you first have to have your own bone marrow cells destroyed. This means having high dose chemotherapy and sometimes 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. The donor is someone whose stem cells match yours. The most suitable donor is usually a close relative such as a brother or sister.

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 at a high risk of developing infections. So your doctor and nurses will give you antibiotics and anti viral drugs to try to prevent them.

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 MDS
  • have a suitable donor

You can talk through the benefits and risks of this treatment with your specialist. They will be able to tell you whether this treatment is an option for you.

Newer treatments

You might have Lenalidomide (Revlimid) to control MDS if you have a particular chromosome change called del5q. You take this orally as a capsule. It works in several ways to suppress the MDS cells, including altering the immune system. It is often referred to as a type of immune modulation therapy.

It has mainly been used to treat lower risk MDS but research is now also looking at it to treat higher risk MDS.

Researchers are looking at other drugs including:

  • panobinostat
  • vorinostat
  • ganetespib

Coping with MDS

Coping with a rare condition can be difficult, both practically and emotionally. Being well informed about your condition and its treatment can help you to make decisions and cope with what happens.

The symptoms of MDS often mean that you feel less able to do everyday things. Talk to your doctor or nurse about any symptoms you have. There may be supportive treatments that can help you.

You may also feel better if you:

  • eat a healthy well balanced diet
  • do some gentle exercise – after checking with your doctor
  • rest when you are tired

Try to work out your priorities each day if you get tired easily. Talk to your family and friends and ask for any help you need. You can find tips about coping with tiredness in the section about fatigue and cancer.

Leukaemia charities also offer support and information to those affected by MDS.

Last reviewed: 
10 Aug 2017
  • Azacitidine for the treatment of myelodysplastic syndromes, chronic myelomonocytic leukaemia and acute myeloid leukaemia (TA218)
    National Institute for Health and Care Excellence (NICE) March, 2011

  • An update on the treatment of myelodysplastic syndromes
    SE. Kurtin and Ep. Demakos
    Clinical Journal of Oncology Nursing. 2010 Jun;14(3):E29-44

  • Guidelines for the diagnosis and management of adult myelodysplastic syndromes
    SB. Killick (and others)
    British Journal of Haematology 2014 Feb;164(4):503-25

  • Hypomethylating agents and other novel strategies in myelodysplastic syndromes
    G. Garcia-Manero and P. Fenaux
    Journal of Clinical Oncology 2011 Feb 10;29(5):516-23

  • Essential Haematology, 7th Edition

    AV Hoffbrand and PAH Moss. 2016.

    Wiley Blackwell

  • Lenalidomide for treating myelodysplastic syndromes associated with an isolated deletion 5q cytogenetic abnormality (TA322)
    National Institute for Health and Care Excellence (NICE) September, 2014

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