MDS treatment and side effects

Updated 12 Oct 2017

The way that myelodysplastic syndromes (MDS) behaves varies from person to person, and on the type of MDS that you have. Both of these factors influence your treatment options. 

The myelodysplasia prognosis, diagnosis and treatment process for all patients will be reviewed by a group of haematologists in the area that you live. This is called a multidisciplinary team meeting. It allows your case to be discussed by many doctors and healthcare professionals in the haematology clinical team. Occasionally, it is necessary to ask for another opinion outside of this group to help either with the diagnosis or to discuss the best form of treatment.

Treatment planning

The treatment that you are offered will depend on the type of MDS you have, your own wishes, your age, your general wellbeing or fitness and the IPSS-R score

Before your treatment starts, your doctor or nurse will explain the benefits and side effects of the chosen treatment so that your consent can be taken. It is likely that you will need to sign a consent form to agree to the treatment. If you are unsure about anything, do ask your healthcare team, as MDS is a complicated disease to understand. The BCSH MDS guidelines recommend that all people who are newly diagnosed with MDS are discussed with a regional or national expert in MDS, given that the disease is rare. You are entitled to ask your doctor if they have done this. The NHS also allows you to ask to see a regional or national expert in MDS if you think that this would help you.

Not everyone needs active treatment, as some people don't have any symptoms. If you are not starting treatment, you will have regular check-ups, which are often referred to as ‘watch and wait’ – or ‘active monitoring’.

Broadly speaking, treatment of MDS includes:

Unfortunately, most people's MDS cannot be cured but MDS can usually be controlled and often improved.

The first question that your doctor will ask themselves is whether there is a treatment option that has a chance of curing the MDS. The only treatments that can possibly cure MDS are 1) a stem cell or bone marrow transplant from another person, or very rarely 2) intensive chemotherapy. 

If a stem cell transplant is an option for you, you will be identified early so that a search for donors can be started and a transplant considered at an early stage.

> Download or order our booklet 'The seven steps: blood stem cell and bone marrow transplants' to learn more about stem cell transplants

Intensive chemotherapy

If you have high-risk MDS, you may benefit from intensive chemotherapy. It is the same treatment that is used to treat acute myeloid leukaemia (AML) and aims to kill a significant proportion of the diseased cells from your bone marrow to allow the bone marrow to work normally again (remission). 

The treatment has a high number of side effects so you need to stay in hospital for four to six weeks for each course. A small proportion of people may be cured by intensive chemotherapy alone, although usually when a donor is available, a stem cell transplant will follow. Achieving remission, even if not able to cure the disease can improve your quality of life (often almost to normal quality) as long as the remission lasts. 

How is intensive chemotherapy given?

Most chemotherapy is given as an infusion into a vein (intravenous), but sometimes as a tablet. It is given as a course or cycle of treatment, where a combination of chemotherapy is given over a number of days followed by a rest period. It is often easier for you to have a Hickman line inserted, which allows all the drugs to be given and blood tests to be taken. This is a line that is carefully inserted into a large vein and can stay in place for all your treatment. The first course of intensive treatment is normally given in hospital but if your bone marrow is then in remission, subsequent treatment can often be started in an ambulatory care / day care unit, although you may have to come back into hospital once the blood counts drop to low levels.

What are the most common side effects from intensive chemotherapy?

The chemotherapy used in MDS is specially designed to kill the cancer cells from the bone marrow, so your blood counts will fall after the chemotherapy and remain low for a number of weeks. Healthy bone marrow cells are also ‘stunned’ in a type of ‘friendly fire’ but can recover better than the MDS cells if remission is achieved. During this time there are serious, sometimes life-threatening side effects, the most common of which are:

  • infections,
  • bleeding, and 
  • anaemia.

Other side effects include: 

  • hair loss,
  • nausea,
  • vomiting,
  • sore mouth,
  • diarrhoea,
  • loss of appetite and taste, 
  • skin and nail changes, and
  • infertility.

Non-intensive treatment

Treatment extending beyond supportive care can be classed as low-intensity, high-intensity or high-intensity with a stem cell transplant. Non-intensive treatment aims to slow the progression of the disease. It may be considered if your blood counts are quite low or falling, or if there are signs that the disease is developing into leukaemia. The idea is to treat the disease with as few side effects as possible, therefore maintaining a good quality of life. These treatments will not cure MDS but may ‘modify’ the disease and are usually given as an outpatient. Some of the treatments are considered novel and therefore may be used as part of a clinical trial.

Hypomethylating agents (HMA)

Hypomethylating agents work on the behaviour of cancer cells at the DNA level, which can turn genes on and off. In MDS, drugs such as azacitidine work to improve bone marrow function and slow the progression to leukaemia. They are currently used in people with high-risk MDS (IPSS Intermediate-2 and High categories) who are not fit enough for a stem cell transplant. Azacitidine is usually given as an injection under the skin. Side effects include:

  • mild nausea,
  • diarrhoea or constipation,
  • skin irritation at the injection site, and
  • becoming more prone to infections (due to lowered blood counts). 

Lenalidomide 

If you have a certain type of MDS called the 5q minus syndrome, or, MDS with deletion of 5q, you may be offered lenalidomide if you are anaemic. This is taken orally as a capsule and works in several ways to suppress the MDS cells, including by altering the immune system. Therefore it is often referred to as a type of immune modulation therapy. On starting the treatment, your blood counts fall before a response is seen. Other side effects include:

  • rashes,
  • fatigue,
  • diarrhoea, and
  • a small increased risk of blood clots.

As lenalidomide can cause birth defects, you must avoid getting pregnant while taking the drug.

Immunosuppressive therapy

In a small minority of people with MDS, the number of the bone marrow cells is unusually low (termed hypoplastic). This is similar to a blood disease called aplastic anaemia. People can sometimes respond to drugs targeted at suppressing the immune system, such as anti-thymocyte globulin (ATG) or ciclosporin.

Allogeneic stem cell transplant

A stem cell transplant, also known as a bone marrow transplant, offers the chance of curing the disease. In an allogeneic transplant, healthy bone marrow or stem cells are taken from another person whose tissue DNA is identical or almost identical to yours. This means the donor is compatible with you. The bone marrow or stem cells are taken from a donor – either a family member (usually a sibling) or an unrelated donor. The donor has a simple blood test to see if they are matched to you – they do not need to have a bone marrow test. The results usually take two to three weeks to be ready.

In the past, only younger people were offered this treatment, but as medical knowledge and experience have progressed, more people can now be considered for a transplant. Reducing the intensity of the treatment before the transplant of donor stem cells means the side effects are less. This approach is called a reduced intensity conditioning (RIC) transplant. About one-third of people who receive this treatment are free of disease over many years but the disease may return (relapse). 

The treatment has many side effects and it is important that the decision to have an allogeneic stem cell transplant is carefully thought through by your healthcare team and yourself. If you are suitable for a transplant, you will be referred to a specialist centre to discuss the benefits and risks of this treatment to you as an individual. Always try to take a family member or friend to the appointments.

> Download or order our booklet 'The seven steps: blood stem cell and bone marrow transplants' to find out more about this treatment.

Supportive care

Everyone with MDS will need supportive care at some stage, either alone or to support the treatment that you are receiving. Supportive care is not directed at the underlying disease but rather at controlling the symptoms and complications caused by the disease. The nature and extent of supportive care needed depend on which blood cells are most affected and exactly how low their blood levels fall. Most people will need blood transfusions at some stage. 

Treating anaemia

Most people (but not all) diagnosed with MDS are anaemic. Although the anaemia is usually not life threatening, it can cause symptoms such as tiredness and shortness of breath. Anaemia may affect your quality of life and if so it will need treatment. Some people continue living a normal or acceptable quality life despite anaemia and will not necessarily need treatment for the anaemia at that stage. The haemoglobin (Hb) level in your blood results will show your level of anaemia. 

Blood transfusions 

Blood transfusions are considered if you have symptoms of anaemia. There is no set haemoglobin level at which a blood transfusion is given, but your doctor will assess your symptoms and you will decide together. How often you have transfusions will vary between different people; some need transfusions every few months, while others need one every couple of weeks. Usually, once you have started having regular blood transfusions, the length of time between transfusions will gradually get shorter. If you find that your symptoms of anaemia come back well before your next transfusion is due, contact your haematology team and discuss whether the interval between transfusions should be shorter, or the number of units of blood increased. This varies between people. 

With every unit of blood you receive from a transfusion, you will receive an excess amount of iron. Over time, this can accumulate in your body and could possibly cause damage to certain organs, like your heart or liver. Because blood transfusions are rich in iron, it is important that you do not take additional iron tablets unless your doctor prescribes them. There is still considerable uncertainty whether too much iron in your body is always harmful. The level of iron in your body should be checked regularly, especially when you are on a regular transfusion program and you may need treatment for the build-up of excess iron. This is called iron chelation. There is currently uncertainty about the benefits of removing iron. Whether you are offered iron or not, iron chelation treatment will depend on the likely benefits versus the likely disadvantages in your individual case. This will be discussed with you before you make a decision start iron chelation. 

Desferal is a drug to treat the build up of excess iron and is given as a continuous subcutaneous injection under your skin by a pump. There are special teams that can teach you how to administer the drug at home. Exjade is another iron chelator and comes in tablet form, making it easier for people with poor sight, problems of finger dexterity or a fear of needles. 

However, in most cases, this is only available for people who cannot manage subcutaneous Desferal or who have serious side effects on Desferal. Both treatments have certain side effects and often need to be continued for a long period of time to be effective. Your doctor can advise you which drug will be best in your situation. Don’t hesitate to discuss your iron levels with your doctor at any time during your treatment.

Growth factors

Blood cell numbers can sometimes be increased by the use of growth factors. Growth factors are like natural ‘hormones’ that stimulate our blood production. We all make these growth factors every day. For example, erythropoietin (sometimes known as ‘EPO’) is a growth factor that increases red cell numbers. Granulocyte-colony stimulating factor (or ‘G-CSF’) increases white cell numbers. Not everyone is suitable for this treatment, and only some people will respond. Your doctor can advise you on your suitability for growth factors.

Growth factors are given as an injection under the skin. The number of injections you will need will vary from person to person. A district nurse can give them at home, or you (or a family member) can learn how to give the injections yourself. The skin around the injection site may become irritated, so it is best to regularly change the injection site. Do talk to your nurses about this and also the common side effects that you may expect.

Platelet transfusions

About half of people with MDS will have a reduced platelet count at diagnosis (thrombocytopenia). The platelets may also function poorly and this means that bruising and bleeding can occasionally be a serious problem in MDS. Platelets can be transfused but because they only last about four days, they are usually only given if you have signs of bleeding. If you have low platelets, it is usually advisable to avoid blood-thinning agents and non-steroidal anti-inflammatory drugs. However this should be discussed with your doctor as there are exceptions where the benefit you will receive from these drugs outweighs the risks.

Most hospitals will not transfuse unless the platelet level drops below 10, and some hospitals will not routinely give platelet transfusion. When you have an infection, are on blood thinners or have suffered from bleeding, you might benefit from having platelets kept at a higher level (having a transfusion earlier than normal). Your doctor or nurse will inform you when this is necessary. 

Antibiotics

It is important for you to understand that people with MDS have a higher risk of developing infections. Antibiotics are not usually given to prevent infections as they cause side effects and may cause the bacteria to become resistant. If you do get an infection, this should be treated quickly with antibiotics, and you may need to be admitted into hospital so that the antibiotics can be given through a vein (intravenous). Most specialist units will have a direct phone number to call for advice in the event of a fever occurring.

Follow-up

For some people, this will only mean infrequent outpatient visits to check if the disease is showing signs of progressing. Sometimes these check-ups can be shared with the GP. For people who are thought to have high-risk MDS or for those who have received active treatment, the outpatient visits may be more frequent. This will be individually tailored to you.

International prognostic scoring system

The International Prognostic Scoring System (IPSS) was developed by analysing information on almost 1,000 people with MDS, who mostly received only supportive care, and determining which factors best predicted disease progression and outcome. This was then used to create a scoring system based on the percentage of blasts in the bone marrow, cytogenetics and the number of cell types affected in the circulating blood. The IPSS is now only used to decide whether patients are suitable for certain treatments that were developed in the IPSS era (1995-2012). Since 2012, all discussions with patients about prognosis should be based on the IPSS-R (see below)

Definitions used in the IPSS:

 

Karyotype

  • Good – normal, deletion of Y chromosome, del(5q), del(20q)
  • Poor – complex (more than 3 abnormalities), chromosome 7 abnormalities
  • Intermediate – all other abnormalities

Cytopenias

IPSS score table

The individual scores for bone marrow blast percentage, karyotype and cytopenias are added together to give the IPSS score. The scores for the risk groups are as follows:

Low        0
INT-1      0.5-1.0
INT-2      1.5-2.0
High        >2.5

The Revised IPSS (IPSS-R)

This scoring system has used the data gathered from patients in the IPSS to further categorise patients into more defined risk groups from over 7,000 untreated people with MDS. The score now has five categories which allow a more accurate idea of expected outcome. Both scoring systems should only be used at diagnosis and not during the course of the disease.

IPSS-R Cytogenetic Prognostic Subgroups

IPSS-R Prognostic Score Values

IPSS-R Prognostic Risk Categories/Scores and Clinical Outcomes

 

PIF memberStandard member