Tests and treatment for myelodysplastic syndromes (MDS)

Myelodysplastic syndromes are a group of blood cancers. They are also called myelodysplasia, myelodysplastic neoplasms or MDS for short.

Diagnosing myelodysplastic syndromes

Your doctor will examine you and ask questions about your medical history and family medical history.

Tests

To diagnose MDS, you have blood tests and bone marrow tests.

The blood tests show how many normal blood cells and how many abnormal or immature blood cells you have.

For a bone marrow test, a doctor or specialist nurse removes a sample of bone marrow to examine it closely. This is usually taken from your hip. Bone marrow is the soft area at the centre of some of our bones. You usually have the test in the outpatient department of the hospital.

As well as looking at the bone marrow cells, your doctor tests for abnormalities in your chromosomes. Chromosomes contain the genetic material inside the cells. These tests are called cytogenetics (pronounced sigh-toe gen-et-ics). Doctors can use blood or bone marrow samples to look for genetic changes.  

Doctors are learning more about MDS all the time. You might have other tests done on the bone marrow to look for other gene changes. You might hear this called next generation sequencing (NGS).

Some people with MDS go on to develop acute myeloid leukaemia (AML). Doctors can look at the results of cytogenetics and NGS tests together. This can help them understand more about the risk of your MDS changing to AML.

You may have other tests if your specialists need more information to confirm your diagnosis. They will explain what these are if you have them.

Treatment for myelodysplastic syndromes

The type of treatment you have depends on:

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

Treatment aims to get the number and type of blood cells in the bloodstream back to normal.

Low risk MDS

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 low levels of blood cells. This is called supportive treatment.

Intermediate or high risk MDS

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

Treatment with a stem cell transplant from a donor is not suitable for everyone. Your specialist will tell you more about this treatment and if it is suitable for you. 

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.

Transfusions

You may have transfusions to treat your MDS. You have a drip containing red blood cells  if your red blood cells are low. 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 reduce 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.

Erythropoietin 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 cell levels. These drugs are given as injections just under the skin (subcutaneously).

Antibiotics

You might also need to take antibiotics to treat infections. When your white blood cell count is low, it is more difficult for your body to fight 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 ciclosporin can sometimes be used to treat a certain type of MDS but this treatment is not suitable for everyone. 

Chemotherapy

Chemotherapy uses 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 could have just one drug or a combination of drugs.

For high risk MDS, you are likely to have chemotherapy similar to that used for acute myeloid leukaemia (AML). The chemotherapy drugs used to treat MDS include:

• azacitidine (Vidaza)
• cytarabine
• fludarabine (Fludara)
• idarubicin (Zavedos)
• melphalan

Intensive treatment with a donor stem cell transplant

When you have a stem cell transplant using another person’s (donor) stem cells, it is called an allogeneic transplant.

This treatment is intensive 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

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 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.

This treatment can have complications. Your specialist will give you more information about how you have it and can talk through the benefits and risks. They will be able to tell you whether this treatment is an option for you.

Lenalidomide

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 has mainly been used to treat lower or intermediate risk MDS. 

Research and clinical trials

Doctors want to find out as much as possible about the many different types of MDS, and how best to treat them.

All treatments must be fully researched before they can be used for everyone. This is so we can be sure that they:

• work
• work better than the treatments already available
• are safe

Cancer Research UK Clinical Trials Database

You can find a clinical trial looking at myelodysplastic syndromes on our clinical trials database. Click on the ‘recruiting’, ‘closed’ and ‘results’ tabs to make sure you see all the trials.

There may not be an open trial and some of the trials have now stopped recruiting people. It takes time before the results are available. This is because the trial team follow the patients for a period of time and collect and analyse the results. We have included this ongoing research to give examples of the type of research being carried out in MDS.

A study team is setting up a record or ‘registry’ of information about a large number of people across Europe with low risk MDS. The aim is for doctors to learn more about MDS and its outcomes.

Drugs to treat MDS 

Doctors in the UK and around the world are looking at new drugs for MDS. They are also investigating new ways to give older drugs. Some trial teams are looking at combining old and newer cancer drugs for people with MDS.

Researchers are looking at treatments to improve low blood levels. They are comparing a combination of drugs sodium valproate, bezafibrate and medroxyprogesterone (VBaP) with another drug called danazol.

They want to know:

• how well treatment works to improve low blood counts
• if treatment reduces the need for blood transfusions
• if treatment increases the length of time people live 
• how treatment affects quality of life  

Other drugs being looked at in clinical trials include:

• quizartinib

• azacitidine

• magrolimab

• MBG453

• BCT-100

• INCB057643

• CCS1477

You need to talk to your specialist if there are any trials that you think you might be able to take part in.

Coping with MDS

We have information about where to find support to help you cope with MDS and having treatment.