The best treatment for you will depend on the type of MDS you have, the risk group you are in, and your general health. Usually, the aim of treatment is to help to improve your blood counts and relieve symptoms.
If you have low-risk or intermediate-risk MDS and don’t have any symptoms, you may not need treatment straight away. Instead, you will have regular check-ups and your blood counts will be monitored. This is sometimes called ‘watch and wait’.
Most people with MDS have treatment at some point to help with the symptoms caused by low blood counts. This is called supportive treatment.
Chemotherapy may be helpful for some people, depending on the type of MDS they have. Sometimes, drugs that affect how the immune system works are used to treat MDS. These drugs are called immunosuppressants.
Some people may have treatment with a targeted (biological) therapy.
A small number of people who are in good general health may be able to have a donor stem cell transplant. The aim of this is to cure MDS.
Supportive treatment
Supportive treatment aims to help with the symptoms of MDS and improve your quality of life.
Transfusions
You may feel tired or short of breath. This is caused by low levels of red blood cells. Or you may have bruising and bleeding caused by low platelets levels. You can have blood or platelet transfusions to help improve these symptoms.
Red blood cells contain iron, so if you have a lot of blood transfusions, the level of iron in your body will increase. Too much iron can be harmful, and you may need treatment to reduce it. Your doctor or nurse will explain more about this treatment if you need it.
Growth factors
It may be possible to boost the number of healthy red and white blood cells in your blood with growth factors.
A growth factor called erythropoietin may be used to increase the amount of red blood cells in your blood and reduce the number of blood transfusions you need. A drug called G-CSF may be given to boost the number of white blood cells.
Both of these growth factors are given as injections under the skin (subcutaneously). Some people may get both drugs depending on their full blood counts.
Reducing your risk of infection
If your white blood cell level is low, you’re at an increased risk of infection. Your specialist nurse or doctor will give you advice about reducing this risk. You may be given drugs, such as antibiotics, to prevent or treat infections.
Chemotherapy
Chemotherapy is the use of anti-cancer (cytotoxic) drugs to destroy cancer cells. It can also be given to help control MDS. The type of chemotherapy you have will depend on your general health and how the disease is affecting you. The aim is to get the bone marrow working normally (remission).
Chemotherapy can be given as tablets, an injection into a vein (intravenously) or an injection under the skin (subcutaneously). You may be given a single drug or two or more drugs together (combination chemotherapy). Chemotherapy may be given as an outpatient or during a short stay in hospital. For some more intensive types of chemotherapy, you may need to stay in hospital for a few weeks.
Azacitidine (Vidaza®) is a chemotherapy drug for people with high-risk MDS who aren’t fit enough to have a stem cell transplant. It can help the bone marrow to work better. It is given as an injection under the skin. Side effects include discomfort at the injection site, sickness and tiredness. It also temporarily reduces the blood count.
Immunosuppressants
Sometimes in MDS, white blood cells called T-lymphocytes affect normal blood cell production. Immunosuppressants work by making T-lymphocytes less active. Immunosuppressant drugs used to treat MDS include anti-thymocyte globulins (ATGs) and ciclosporin.
ATG is given as a drip through a vein. It is given in hospital because it can sometimes cause allergic reactions. Ciclosporin can be taken at home as capsules or a liquid.
Targeted (biological) therapy
Some people with isolated del 5q MDS may be given treatment with a targeted therapy called lenalidomide (Revlimid®). Lenalidomide works on the immune system to target the affected blood cells.
Stem cell transplant (from a donor)
A small number of people with MDS may be able to have a stem cell transplant from a donor, also called an allogenic transplant.
You have high doses of chemotherapy, and sometimes radiotherapy. The stem cells that have been destroyed by the treatment are then replaced. The replacement stem cells are from a donor. A donor can be a brother, sister or an unrelated person who is a match for you.
This treatment is only suitable for some people with MDS. Your specialist will discuss the possible benefits and disadvantages of this treatment with you.
Clinical trials
There are also newer treatments available that may be used to help to control MDS.
You may be invited to join a clinical trial that is looking at new ways of treating MDS. You can talk about this with your haematologist.