Myelofibrosis (MF) treatment and side effects
In this section we talk about the specific treatments used for MF. You might also want to read about how blood cancer treatment is planned and managing your treatment.
We also have some important information on staying safe if you’ve got blood cancer, covering things like risk of infection and vaccinations.
The main aim of treatment is to control any symptoms you have. If you’ve been diagnosed with MF and don’t have any symptoms, you may not need to start treatment for a while.
The treatment you have will for MF depends on a variety of factors, including:
- your blood counts
- the symptoms you have
- your overall fitness.
You should be monitored for cardiovascular risk factors such as diabetes, high cholesterol, high blood pressure and smoking, as these should be addressed as effectively as possible.
If you don't need to start treatment, you'll be monitored with regular check-ups and blood tests. This is known as 'watch and wait'. While this might seem strange, there’s no evidence to show that treating people who don't have any symptoms has any impact on their outcome. It also means you don’t get side effects from unnecessary treatment.
We have more information on watch and wait.
If you have severe anaemia you’ll need regular blood transfusions, usually every one to three months. These can be carried out during a single day and you would need to stay in hospital overnight. You might also have platelet transfusions if you have low platelets and you’re having unusual bleeding or bruising.
Find out more about blood and platelet transfusions.
Ruxolitinib is a type of drug known as a JAK2 inhibitor. It is recommended for people with an enlarged spleen or troublesome symptoms of MF. It can reduce the size of your spleen, improve symptoms and improve your overall prognosis (the forecast for your future). It is taken as tablets, twice a day, for as long as it is working.
Possible side effects of ruxolitinib include anaemia, a low platelet count, a low white blood cell count and increased risk of infections.
Your doctors will monitor your response to ruxolitinib. They may need to change the dose or stop ruxolitinib if it’s not working well for you.
If your platelet count is high or you have other symptoms such as weight loss or sweats, you may be given tablets called hydroxycarbamide (or hydroxyurea) to take. This is a mild form of chemotherapy and works by directly preventing the production of red blood cells. Hydroxycarbamide is the most common chemotherapy drug used to treat MF.
You might get some side effects from this treatment. These might include more infections than normal, diarrhoea or constipation. Your healthcare team will be able to help you manage side effects like this.
Hydroxycarbamide is a very safe treatment. However, there’s a theoretical risk that it may increase the risk of MF transforming into acute myeloid leukaemia (AML) if it’s used as a long-term treatment. For many people, it’s felt that the benefits of the treatment outweigh any potential small risk involved.
Drugs called JAK2 inhibitors are now available to treat MF. These can reduce the size of your spleen, improve symptoms and your overall outlook. However, JAK inhibitors may worsen anaemia.
The length of time you take the drug for depends on how well it works and any side effects you get from it. Your doctor can discuss if these medications would be appropriate treatment for you.
Thalidomide is a targeted therapy to treat MF. Thalidomide can cause birth defects, so it shouldn’t be given to pregnant women. People taking thalidomide who are sexually active should use a barrier form of contraception as some hormonal methods of birth control (such as the pill) can be made less effective by thalidomide.
You may also get side effects from this treatment such as feeling tired or drowsy, constipation and numbness or tingling in your hands and feet (peripheral neuropathy).
The length of time you take the drug for depends on how well it works and any side effects you get from it. The use of thalidomide is becoming less common in the UK.
You may be given danazol to help improve anaemia (low number of red blood cells). The length of time you take the drug for depends on how well it works and any side effects you get from it.
A stem cell transplant aims to give patients healthy stem cells, which then produce normal blood cells.
It isn’t a suitable treatment for most MF patients due to the risks associated with the procedure. For a minority of patients – especially those whose disease is progressing more quickly – a transplant may provide a cure, but there isn’t enough evidence to be sure of this yet.
There are two main types of stem cell transplant:
- autologous or autograft – this uses the patients’ own stem cells
- allogeneic or allograft – this uses donor stem cells and is a high risk procedure.
We have more information on stem cells transplants.
An enlarged spleen may cause you problems by becoming painful. It may also cause anaemia.
To treat an enlarged spleen, you may receive local radiation. This would usually reduce the size of your spleen for a period of time, from anything between a few months to a couple of years.
Another option may be to have an operation to remove your spleen (a splenectomy). There can be complications after a splenectomy. Your doctor will discuss the pros and cons with you to help you decide if the procedure is right for you.
Watch and wait
If you’re on watch and wait, learn what it means and find ways to cope.