Myelodyspastic Syndromes (MDS) Treatment Options
Once the diagnosis of MDS has been made, you and your doctors will decide on the best treatment for you. The treatment that you are offered will depend on the type of MDS you have, your own wishes, your age, your general well-being or fitness and the IPSS-R score.
Broadly speaking, treatment of MDS includes:
- supportive care – this aims to control the symptoms of MDS
- non-intensive treatment – this treatment tries to slow down the progression of MDS and improve your blood counts
- intensive chemotherapy – this involves giving high doses of chemotherapy in hospital
- stem cell transplant - also known as bone marrow transplant
New treatments are being developed for MDS. The process by which a new treatment is tested and evaluated is called clinical trial. Clinical trials aim to gather the evidence necessary to make a decision about changing standard practice. There are many drugs currently being tested. Your doctors will inform you if you qualify to take part in these trials.
Despite the great progress made in fighting MDS, unfortunately most patients’ MDS cannot be cured - however, MDS can usually be controlled and often improved.
This booklet has been written to help you understand more about MDS. Download it and learn more about MDS and the different options for treatment in Chapter 7.
Supportive care is not directed at the underlying disease but rather at controlling the symptoms and complications caused by the disease.
Supportive care includes:
- Blood transfusions, to treat anemia
- Growth factors, which are natural ‘hormones’ that stimulate blood production, such as erythropoietin (also known as ‘EPO’)
- Platelet transfusions
- Antibiotics, to treat an infection quickly, as MDS patients have a higher risk of developing infections
Non-intensive Care - Active Treatment
This includes a number of drugs, such as azacitidine, which aim to slow the progression of the disease. They are active treatments, but they are known as "low intensity" or "non-intensive" treatments as they do not have the side-effects normally associated with chemotherapy.
Non-intensive care includes:
- Hypomethylating agents (HMA), which work on the behaviour of cancer cells at the DNA level, e.g. drugs such as azacitidine
- Lenalidomide, only suitable for 5q minus syndrome patients, suppresses the MDS cells, by altering the immune system
- Immunosuppressive therapy, only suitable for those patients whose number of bone marrow cells is unusually low (hypoplastic). Anti-thymocyte globulin (ATG) or ciclosporin are immuno-suppressants.
This treatment is the same as that used to treat acute myeloid leukaemia and aims to kill a significant proportion of the abnormal cells in the bone marrow to allow the bone marrow to work normally again (remission).
Achieving remission, even if not a cure for the disease, can improve your quality of life (often almost to normal quality) as long as the remission lasts.
The treatment, however, has a high number of side effects so patients 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.
Stem Cell Transplant
A stem cell transplant, also known as a bone marrow transplant, offers the chance of curing the disease. About one third of patients who receive this treatment are free of disease over many years but the disease may return (relapse).
It's called allogeneic stem cell transplant. Allogeneic means that the transplanted cells are coming from a donor. Healthy bone marrow or stem cells are taken from another person whose tissue DNA is identical or almost identical to yours, so the donor is compatible with you.
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.
For a small group of patients, the long-term and late effects of the transplant can have a very serious impact on their general health and wellbeing. As a result good quality of life can be difficult to achieve. This book, The Next Steps hopes to address some of these in more detail to help you understand more about the transplant journey.