Treatment will depend on your type of MDS, your risk group, and whether you have any other health conditions.

If your MDS is low risk and you do not have any symptoms it is likely that you will not need any treatment at first. You will be monitored with regular blood tests to check how the condition is behaving. We call this watch and wait.

Very low to intermediate risk disease

For patients who have very low to intermediate risk disease, symptoms of MDS can be controlled with a combination of the following treatments:

  • injections of growth factor drugs such as erythropoietin (which increases red blood cell count) or G-CSF (which increases white blood cells) to encourage your bone marrow to make more blood cells
  • a blood transfusion – the drip may contain red blood cells or platelets, depending on which cells have been affected
  • drugs to get rid of the excess iron in your blood (which builds up after a lot of blood transfusions)
  • antibiotics to treat infections, if your white blood cell count is low)

Drugs such as anti-thymocyte immunoglobulin (ATG) and ciclosporin reduce the activity of the immune system, allowing your bone marrow to make blood cells. It can also help to control symptoms.

However, these drugs are not suitable for everyone and work best in young people and those who don't have a chromosome change associated with their condition.

Intermediate to very high risk disease

MDS patients may present with lower risk disease and then over time evolve to higher risk disease. Alternatively, MDS patients may present at a time when their disease is already in a higher risk category. We have several treatment options available for patients with higher risk disease:

Azacitidine

Azacitidine is approved by National Institute for Health and Care Excellence (NICE) for the treatment of MDS patients who are not eligible for a stem cell transplant. Azacitidine is a type of chemotherapy that works by altering the DNA of the MDS cells (called a hypomethylating agent). It is given by injections under the skin for 7 days each month. The treatment is given as an outpatient.

This involves taking drugs that destroy the immature blood cells by disrupting their growth. The drugs are usually given by injection and require you to stay in hospital for a few weeks.

If you MDS that is at high risk of transforming into AML, your chemotherapy treatment will probably be similar to that used to treat AML. Read about the treatment of AML.

You will probably have one or a combination of the following chemotherapy drugs:

  • cytarabine
  • fludarabine
  • daunorubicin
  • cytosine arabinoside

The only way to cure MDS is to have intensive treatment with a stem cell transplant from a donor – but this isn't suitable for everyone.

A stem cell transplant will generally only be offered if you are young and in reasonably good health (apart from your MDS), as it's a very intensive treatment.

It helps if you have a suitable donor in your family (a close relative, such as a brother or sister), although in some cases, it's possible to have a stem cell transplant using an unrelated donor with a matching tissue type.

Treatment involves destroying your own bone marrow cells with chemotherapy and sometimes radiotherapy, before having stem cells from a donor fed into your bloodstream via a drip.

Lenalidomide is a drug that modulates the immune system. This drug appears to have a very specific effect in patients with MDS who have an abnormality in the chromosomes of the bone marrow called an isolated deletion 5q.

Lenalidomide is taken by mouth and is given for 21 days out of every month.

Clinical trials

The treatment of MDS is constantly evolving and new drugs are being tested all the time.

We are currently a number of clinical trials for new therapeutic agents in MDS:

MDS-001 and MDS-006

These studies are designed for patients with intermediate or higher risk MDS and compares the current standard of care in these patients (azacitidine - Vidaza) with azacitidine in combination with a new drug called duvalumab which works by enhancing the immune systems ability to “see” MDS cells. In MDS 006 patients who have not improved with the licenced injectable form of azacitidine or are unable to tolerate this formulation will be eligible to receive CC-486 +/- durvalumab. CC-486 is an oral preparation of azacitidine which has been shown to have effect in low risk MDS to date. The study is taking place in many countries around the world. In the UK Dr Beth Payne is the chief investigator for MDS-006.

For further information please contact: Alexandra.Brownsdon@uclh.nhs.uk

UCLH has a phase I clinical trial unit. Haematology trials are run by Dr Rakesh Popat. Phase I trials are usually for patients that we do not have any further licenced treatments available for. This is because these are often very new drugs that we do not know whether they will be effective or not. Several of these studies are open to patients with MDS – please see the CRF website for further details