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Myelodysplastic syndromes treatment

How is MDS treated?

The treatment chosen for your disease depends on several factors including the exact type of MDS you have, your age, other prognostic factors, and your general health. Remember that no two people are the same. In helping you to make the best treatment decision, your doctor will consider all the information available including the details of your particular situation. Your doctor will be able to discuss with you all of the treatment options suitable for you.

Watch and Wait/Active Monitoring

Many people, particularly in the early stage of disease, remain very well and live a relatively normal life. They may not need to start any treatment as they don’t have any symptoms. Your haematologist may recommend regular check-ups to keep an eye on your health. You might need to see your GP for blood tests. How often will depend on any changes in your blood counts and your general health. This is called ‘watch and wait’.

Supportive care

Supportive care is the mainstay of treatment for the majority of people with MDS. This involves making every effort to improve your quality of life by relieving any symptoms you might have and by preventing and treating any complications that arise from your disease or treatment. If symptoms of anaemia are interfering with your normal daily activities, your doctor may recommend that you have a red blood cell transfusion. Over time, repeated red blood cell transfusions can lead to a build-up of high levels of iron in the body. Your doctor will be able to tell if this is happening from a simple blood test. If needed, a special drug can be given which binds the iron and helps to safely remove it from the body. Antibiotics and in some cases, the use of growth factors, are all important elements of supportive care. Growth factors are natural chemicals in your blood that stimulate the bone marrow to produce different types of blood cells. Growth factors are given as an injection under the skin (subcutaneous).


Chemotherapy literally means therapy with chemicals. Many chemotherapy drugs are also called cytotoxics (cell toxic) because they kill cells; especially ones that multiply quickly like cancer cells. In general, chemotherapy is only used in MDS in situations when there is a need to control a rising white cell count or if the MDS is transforming or has transformed into leukaemia. Chemotherapy is also given to treat a subtype of MDS called chronic myelomonocytic leukaemia (CMML), which is characterised by a higher than normal white cell count in the blood. DNA Methyl Tranferase Inhibitors, a type of low dose chemotherapy given via injection, has shown benefits in patients with higher risk MDS.

The aim of chemotherapy is to reduce the number of blast cells in your bone marrow and, by doing so, allowing the remaining normal stem cells to make normal red blood cells, white blood cells and platelets. Chemotherapy may be administered in three different situations in MDS.

  • Low-dose oral chemotherapy for CMML

Low doses of oral chemotherapy (chemotherapy that is taken by mouth) can be very effective at controlling a high white cell count. It is usually very well tolerated and does not usually cause nausea (feeling sick) or significant hair loss, although it can cause dry skin. The dose of the chemotherapy drug can be adjusted to the response of the white cells and also the response of other blood cells such as red cells and platelets. Blood counts are monitored frequently while you are receiving chemotherapy.

  • Low-intensity chemotherapy for high-risk MDS/AML

Chemotherapy can be used to control a rising blast count in the peripheral blood. This is often seen when MDS is transforming to acute leukaemia. The aim of this treatment is to control the leukaemia while avoiding any severe side effects from chemotherapy. The main treatment for MDS is a type of chemo called a hypomethylating agent. This low-intensity chemo is different to standard chemotherapy because it works on the genes that affect how normal blood cells develop. It is hoped that this will enable you to have a reasonable quality of life and continue living at home, although visits to the chemotherapy day centre or clinic may be necessary two or three times a week.

  • Standard-dose chemotherapy for high-risk MDS/AML

People who have MDS that is transforming, or has transformed, into acute myeloid leukaemia may benefit from standard anti-leukaemia therapy if they are fit enough. Not everyone is suitable for this form of treatment, especially if they are elderly or frail. Unfortunately, even if a complete remission is achieved, most patients will relapse and the leukaemia will reappear, usually within a year. The decision to have this type of treatment needs to be discussed by you and your family in detail with your doctor. This treatment is given in hospital and the side effects can be more severe.

Stem cell transplantation

Stem cell transplantation (also called a bone marrow transplant) using a suitably matched donor is the only potential cure for MDS. However, this treatment carries significant risks and is only suitable for a very small minority (<5%) of younger patients with MDS. A newer approach in stem cell transplantation involves using less intensive doses of chemotherapy. This approach may be suitable for selected patients older than 50. More moderate doses of chemotherapy are used to destroy enough abnormal stem cells in the bone marrow and suppress the patient’s immune system enough for it to accept the new, donated stem cells. This is called a reduced intensity transplant, mini transplant, or a mini-allogeneic (mini allo) stem cell transplant.

Current and evolving drug therapies

There are several new approaches being developed for the treatment of MDS. These include new chemotherapy drugs, targeted therapies, biological modifiers and drugs that harness the power of the immune system to help fight disease. Some of these drugs are available in Australia, while others are only available on clinical trials.


Last updated on August 9th, 2022

Developed by the Leukaemia Foundation in consultation with people living with a blood cancer, Leukaemia Foundation support staff, haematology nursing staff and/or Australian clinical haematologists. This content is provided for information purposes only and we urge you to always seek advice from a registered health care professional for diagnosis, treatment and answers to your medical questions, including the suitability of a particular therapy, service, product or treatment in your circumstances. The Leukaemia Foundation shall not bear any liability for any person relying on the materials contained on this website.

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