Treatments for blood cancers and blood disorders

Types of treatment

Watch and wait (active monitoring)

This means keeping an eye on your condition and only starting treatment if there are signs that your condition is developing further. You will continue to have regular tests and check-ups (1). Your doctor may call this active monitoring or active surveillance.

Your doctor may suggest this approach if you do not have symptoms or if they are not troubling you too much. With some conditions that tend to develop slowly, it is better to keep treatment in reserve for when you really need it (1). Yiuyr doctor will only do this if there is no proof that early treatment will help your condition.

All treatment has side effects so there is no benefit in exposing you to them if the treatment would not help in the long term.

Chemotherapy

This is a type of treatment with anti-cancer drugs (2). It is used to treat nearly all types of leukaemia, lymphoma and myeloma blood cancers.

Milder forms of chemotherapy, usually tablets, are also used to treat some blood disorders where there are too many blood cells.

Blood cells need to be constantly replaced with new cells. All types of body cells grow by multiplying – a single cell dividing into two new cells. Cancer cells are abnormal and they divide much more often than they should (3). In blood cancers, it is particular types of white blood cells that grow out of control. In some blood disorders, blood cells also grow too quickly.

When cells are in the process of dividing, they are more vulnerable to chemotherapy drugs. So chemotherapy works by killing these cells that are dividing more often than normal (3).

Treatment

There are many different types of chemotherapy drugs. You may have treatment with one drug or a combination of several different drugs. Some can be swallowed as tablets, but others have to be given through an IV drip into your bloodstream (2).

In some types of leukaemia, there is a risk that cancer cells could reach your brain. Your doctor may suggest ‘intrathecal’ treatment to try to prevent this (4). It means giving chemotherapy through an extremely thin tube put into your spine, in the same way as an epidural anaesthetic.

Side effects

Side effects vary between different chemotherapy drugs. They also vary between people. Some side effects are very common: just about everyone gets them (5). Others are less common and you would not know whether you are going to get them or not until you have treatment (6). The dose of the drugs also affects the side effects (5).

Just about all chemotherapy drugs will lower your resistance to infection. Other common side effects include (6):

• tiredness
• sickness or diarrhoea
• sore mouth
• bruising or bleeding
• hair loss
• loss of fertility (you cannot get pregnant or father a child).

There are many other possible side effects, too many to list here. Your own doctor will be able to tell you which are most likely with the chemotherapy drugs you are having.

Most side effects are temporary; they will go when you finish treatment. There are medicines that can control or reduce many of them. But the effect on fertility can be permanent, so talk to your doctor before you start treatment if you are concerned about this (6).

Find out more: Cancer Research UK’s cancer drug finder gives more information about the side effects of individual drugs.

Targeted therapies (biological therapies)

This is a group of relatively new types of treatment, sometimes also called biological therapies (9), are used to treat blood cancers and sometimes myelodysplastic syndrome (7,8). The drugs are manufactured to target particular proteins carried by cancer cells (7).

These drugs work in different ways, depending on the receptor they are targeting (7). The drugs:

• may stop the cells dividing and growing (10)
• may look for cancer cells and then kill them when they find them (7)
• enable the immune system to find and kill them (7).

Some, particularly those used to treat myeloma, stop the cancers from growing blood vessels that supply the tumours with oxygen and food (7).

Targeted therapies are grouped in different ways, depending on how they work and on the type of drug they are. This does not really matter when you are having the treatment, but you may hear your doctors talk about growth blockers, tyrosine kinase inhibitors, monoclonal antibodies or growth inhibitors (7).

Find out more: Cancer Research UK. Types of targeted cancer drugs.

Treatment

This varies, depending on the drug you are having. Many targeted therapies are swallowed as tablets, others you receive through a drip (IV) into your bloodstream (10). Or you may treat it with a subcutaneous injection just under the skin (11).

Side effects

The side effects of targeted therapies vary a lot, depending on the drug you are having. They often cause tiredness, sickness or diarrhoea and flu-like symptoms (10). Like chemo, they can increase your risk of picking up infections (12). Cancer growth blockers may cause skin rashes and a sore mouth (10). Targeted therapies that prevent cancers growing blood vessels can cause numbness or pins and needles in fingers and toes (12).

Most of these side effects are temporary and will go away when you finish treatment (10). Some, such as pins and needles, can last longer and may be permanent in some cases.

Find out more: Cancer Research UK's cancer drug finder gives more information about the side effects of individual drugs.

Immunotherapies

These are treatments that help your immune system to find and kill cancer cells. Some of these are also targeted therapies (10). The most common of these used for blood cancers is rituximab. It looks for cells with a particular protein on the surface and then sticks to them (13). This makes it easier for your immune system to find and kill the marked cells (13).

If you have the blood disorder aplastic anaemia, you may have treatment to dampen down your immune system. This is usually a drug called ciclosporin with or without antibody infusions, given through a drip (14).

There is another type of immunotherapy - CAR-T therapy.

Treatment

You are most likely to have immunotherapy through an IV drip into a vein, or as a subcutaneous injection just under the skin. Before you have rituximab as an injection, your doctor is likely to want you to have it through a drip first, to make sure you do not have a reaction to it (13).

For treatment to dampen down your immune system, you have antibody therapy through a IV drip into your bloodstream and ciclosporin by mouth, as a capsule or liquid that you drink (15-17).

Side effects

Immunotherapies can lower your resistance to infection. They may also cause tiredness, sickness or diarrhoea, bruising or bleeding and flu-like symptoms, such as aching and a raised temperature. It is also possible to have an allergic reaction when you first have treatment. You will have medicines beforehand to try and prevent this. Your nurses will monitor you for any signs, which can include a high temperature, chills, difficulty breathing and sickness (13).

These side effects are temporary and will go away when you finish treatment.

The side effects of treatment to dampen down your immune system include:

• allergic reaction causing a high temperature
• sickness
• headache
• aches and pains
• swelling
• itchy rash
• diarrhoea, and
• blurred vision (14,18).

Ciclosporin can also cause tiredness, dizziness and kidney problems (16,17). It may also cause increased face and body hair, but this goes back to normal when your treatment finishes.

Find out more: Cancer Research UK's cancer drug finder gives more information about the side effects of individual drugs.

Blood stem cell transplants

This type of treatment is widely used to treat lymphomas, leukaemias and some blood disorders (19). The aim of the treatment is to replace faulty blood cells with healthy ones, usually from a donor. There is a type of transplant that involves collecting your own stem cells and giving them back to you. This is called an autologous transplant (20).

Mostly, stem cell transplants involve having cells from a donor, called an allogeneic transplant. This may be a close relative whose blood cells and bone marrow closely match your own. If you do not have a related donor, you may have a ‘matched unrelated donor’ or MUD transplant (20).

Treatment

The first step in having a stem cell transplant is to get rid of your own bone marrow, which is producing faulty blood cells. To do this, you have chemotherapy, with or without radiotherapy. You then have your new stem cells through a drip into your bloodstream (20).

The new stem cells find their own way through your bloodstream and into your bone marrow, where they start growing and producing new blood cells (20). This process is called engraftment (21). While you are waiting for this to happen, you will be at a high risk of infection. So to protect yourself, you will need to be in hospital in isolation (22,23). You may be in hospital for several weeks (20).

This is quite a difficult treatment to get through, so you have to be fit enough for your doctor to consider it (20).

Side effects

The side effects of a stem cell transplant are related to the chemotherapy, and sometimes radiotherapy, that you have to prepare for your new stem cells. These include:

• sickness
• diarrhoea
• a sore mouth
• loss of appetite
• hair loss, and
• loss of fertility (24).

Other side effects are related to your bone marrow having been killed off temporarily. Lack of white blood cells increases your risk of infection. Low red cell count causes anaemia, resulting in tiredness and breathlessness. A low platelet count stops your blood from clotting properly, increasing the risk of bleeding and bruising. For example, you may have nosebleeds, a skin rash and bleeding gums. There is also a risk of donor cells damaging your own body tissues. This is called ‘graft versus host disease’ (GvHD) (24).

We have more detailed information about stem cell transplants, including:

• preparing for a transplant
• stem cell collection
• having your transplant
• follow up care after a transplant
• graft versus host disease (GvHD).

Find out more: Donating blood stem cells.

Radiotherapy

This is a treatment that uses X-ray radiation to kill cancer cells (25). It is not used for blood disorders and is not often used for blood cancers. But you may have it for some types of lymphoma (26).

If you have acute leukaemia you may have radiotherapy to your brain if your doctor suspects that leukaemia cells may have spread there (27).

You may have total body irradiation (TBI) before a stem cell transplant (20,21). This means having radiotherapy to your whole body. The aim is to kill off the blood stem cells in your bone marrow before you have your transplanted stem cells to replace them (21).

Unlike drug treatments, radiotherapy only treats the area of the body it is aimed at. As with chemotherapy, dividing cells are more likely to be killed, including cancer cells. Cells in the bone marrow are rapidly dividing so they are also more likely to be killed (28).

Treatment

You will need to have a planning appointment before you start radiotherapy treatment. This is to work out exactly where to aim the treatment and the dose you will need (28).

Having radiotherapy is similar to having an X-ray: you can’t feel it. The machine may rotate around you to give treatment from different directions (29). You usually have a course of treatments daily or every few days. You are most likely to have a treatment once a day, Monday to Friday (26,29). Radiotherapy for lymphoma is usually aimed at a group of lymph nodes in one area of the body (26).

If you are having radiotherapy to the brain, you will need to have a mask made. This fits over your head and keeps it completely still while you are having treatment (29).

If you are having TBI before a stem cell transplant, you may have a single treatment or treatments twice a day for a few days (23).

Side effects

The side effects of radiotherapy depend on the area of the body being treated (25,29). It can cause hair loss in the treatment area (25). Over a course of treatment, you may develop sore skin, a bit like sunburn. You may be increasingly tired as the course goes on. If your head is in the treatment field you may have a dry, sore mouth (29). Treatment to your stomach or abdominal area can cause sickness or diarrhoea (25,29). You may have anti-sickness medicines to take shortly before you have treatment (21).

If you are having TBI you may have all these side effects (21). You will also be very much at risk of infection until you have your stem cell transfusion and the transplant has taken (23).

Find out more: Donating blood stem cells.

Radiotherapy can have some long-term effects. This depends on the area of the body being treated. If you have treatment that includes the testicles or ovaries, it can cause infertility (28). In women, this can lead to early menopause and vaginal dryness (30)). Treatment that includes the eye in the treatment field, including TBI, can cause clouding of the lens of the eye (cataracts). Treatment that includes the lungs can cause coughs and shortness of breath (28).

Surgery

This is rarely used to treat blood cancers or blood disorders. But some conditions cause a very enlarged spleen. These include hairy cell leukaemia, beta thalassaemia and primary myelofibrosis (31,32). If your spleen is causing discomfort or pain, your doctor may suggest removing it (31 )).

Treatment

Having your spleen removed requires major surgery with a general anaesthetic. It is sometimes done using keyhole surgery, but you may need open surgery, with a larger incision. There is more detailed information on the NHS website about having your spleen removed.

Side effects

There will be the usual issues of getting over surgery, which will take a few weeks. A long-term effect of having your spleen removed is an increased risk of infection. Your doctor will recommend that you have some extra vaccinations, such as against pneumonia. You will also need to have a flu vaccine every year (33). Some doctors prescribe antibiotics to prevent infection and you may need to take these for a year or more. When not on daily antibiotics, you will have a prescription to keep at home. Then you can start taking antibiotics as soon as you have any sign of infection (34).

Tretinoin

Tretinoin is similar to vitamin A. It is also known as ATRA or Vesanoid. Tretinoin is used to treat a very particular type of acute myeloid leukaemia alongside chemotherapy (35).

Treatment

Tretinoin comes as a capsule that you swallow (35). You take it twice a day for up to three months.

Side effects

Tretinoin has quite a few possible side effects (35). As with any drug, you may or may not have them and can’t tell until you start taking it. Some of the side effects are similar to those of chemotherapy so you may not know which drug is causing what.

Among the side effects of Tretinoin are (35):

• sickness
• diarrhoea or constipation
• difficulty sleeping
• mood changes
• anxiety or depression
• headaches
• dizziness
• loss of appetite
• skin rashes, and
• bone pain.

Talk to your doctor if you are concerned about any side effects caused by taking Tretinoin.

CAR-T (T cell therapy)

This is a largely experimental treatment, although it has been approved for adults with some types of lymphoma and children and young people with a particular subtype of acute lymphoblastic leukaemia (36,37).

The treatment involves collecting some of your own white blood cells, called T cells. These are then genetically engineered so that they make a protein called CAR (chimeric antigen receptor). This protein is designed to find and kill the leukaemia or lymphoma cells (38). Chimeric means it is a protein that has been engineered to carry out two functions - attaching to cancer cells and then activating the T cell to attack them.

Treatment

CAR-T cell therapy takes a few weeks. You have the cells collected from your bloodstream through a drip in each arm, a procedure known as apheresis (38). Your blood flows out of one arm, through a machine that collects the cells and then back into the other arm.

The T cells are modified in a laboratory to make the CAR protein. The modified cells are grown in the laboratory until there are enough of them to give back to you through a drip (38).

Usually, you will have chemotherapy before you have the CAR-T cells. This is in order to kill off some of your T cells before you have the modified ones returned to you (38).

Side effects

It is possible to have an allergic reaction when you have the CAR-T cell infusion. You will have medicines beforehand to try and prevent this. Your nurses will monitor you for any signs, which can include a high temperature, chills, difficulty breathing and sickness (38).

The treatment can cause a collection of symptoms called cytokine release syndrome. This happens within a couple of weeks of having the CAR-T cell infusion. It may cause a high temperature, dizziness and difficulty breathing (38).

CAR-T cell therapy can also be neurotoxic. This can cause headaches, confusion, drowsiness, difficulty speaking and fits (seizures). Your doctors and nurses will monitor you very closely for any of these signs and treat them straight away if necessary. They do sometimes go away on their own (38).