The main aims of the treatment are to control symptoms of the disease, including low blood counts and the size of the spleen. Additional treatments may be considered to control the disease as it progresses.
Treatment to control symptoms:
Blood Transfusions: We use blood transfusions to treat anaemia. If your platelet count is very low or there are problems with bleeding, we use platelet transfusions .
Danazol: Danazol is a hormone that is made naturally by the body. This hormone can stimulate the production of red cells and can sometimes make the haemoglobin level rise so that blood transfusions are not needed. This drug is given orally. Side effects include weight gain due to fluid retention and a slight increase in facial hair growth in women. It can also affect the liver or kidneys.
Erythropoetin: Sometimes anaemia is main problem for MF patients. In this case treatment with erythropoetin (EpO) can be effective. EpO is the hormone naturally produced by the body that stimulates the bone marrow to make red cells. This drug is given by injection under the skin.
Aspirin: Low-dose aspirin (75mg once a day) may be given if the platelet count is high and there is a risk of blood clots (thrombosis). Aspirin has side effects, as it can cause bleeding and ulcers in the stomach and gut. Sometimes we use drugs similar to aspirin such as dypyrimadole and clopidogrel.
Splenectomy/Splenic Irradiation: If the main symptom is abdominal pain and discomfort due to a very large spleen, or if the need for blood and platelet transfusions is very high, or if there is no response to medical treatment, it may be beneficial to remove the spleen. There are risks of bleeding and infections with this operation. Occasionally radiotherapy can be given to try to reduce the size of the spleen.
Treatments to control disease progression:
There are some treatment options that try to reduce the rate at which the disease progresses. These include:
- Hydroxycarbamide
- Busulphan
- Thalidomide and prednisolone
- Interferon Alpha
- Low-dose cytarabine
- Bone marrow transplants (BMT)
Hydroxycarbamide: This drug interferes with cell metabolism. It is a daily treatment and blood counts need monitoring every two to three months.
The side effects include some darkening of skin, mouth and leg ulcers, and sometimes stomach problems. Hydroxyurea can damage cells and may affect fertility. Women who are pregnant or those trying to conceive should not use take this drug. There is a small chance that hydroxyurea may increase the risk of acute leukaemia.
- Busulphan We use busulphan hen there are side effects with hydroxyurea, or when it is difficult to take hydroxyurea tablets. Like hydroxurea, busulphan affects the bone marrow directly and can lead to a fall in the blood counts. It is usually given in short courses with regular monitoring. Busulphan can cause lung problems as a side effect. These tablets can damage fertility as well as damaging the bone marrow, and are known to increase the chance of the disease changing into acute leukaemia.
- Thalidomide & Prednisolone Thalidomide combined with steroids can occasionally help with the anaemia and can be very effective in some patients. There is a suggestion that in some patients with early MF, it may reverse early fibrous deposits. Thalidomide is a medication that works by reducing the blood flow to the marrow and stopping the fibroblast cells from causing the scarring. It can cause nerve problems in some patients, problems with sleep and also constipation. This medication is given in low doses for MF so it is generally reasonably well tolerated. The main side effects of steroids are weight gain, increased appetite, mood swings and gastric irritation. If used long term this treatment can lead to bone thinning (osteoporosis) and increased tendency to diabetes.
- Interferon Alpha Interferon Alpha is a natural agent that reduces the production of bone marrow cells and is given by injection several times a week. The side effects are mainly flu-like symptoms, hair loss, depression, and liver and thyroid changes. It does not increase the risk of leukemia and can be used in pregnancy. There is a suggestion that in early cases of MF, interferon alpha can reverse some of the fibrosis and scarring.
Low dose cytarabine Cytarabine is given by injection twice a day. It too affects the bone marrow directly by interfering with the production of cells. We usually give this medication if the disease is progressing quickly or the spleen is very large and causing discomfort. It is given in short courses and needs regular monitoring.
- Bone Marrow Transplants (BMT)
The only treatment that can cure MF is a bone marrow transplant from a sibling or from an unrelated volunteer donor whose marrow cells match the patient well. Blood samples from the patient and the potential donor can be taken to look at their tissue typing to see if they match.
Bone marrow transplants have significant complications in the phase leading into the transplant itself. A BMT also causes long-term problems that may develop after the procedure due to rejection. These risks are why BMTs are reserved for MF patients with a poor prognosis, or those who have not responded to other treatments. Some patients can have bone marrow cells collected and stored for use later if they are needed, but this process is experimental at this stage.
If you'd like to read more about treatments, both conventional and alternative, please click here to go to the Treatments main page |
