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Medical options for blood cancers and disorders

A stem cell transplant is one of many possible treatments for blood cancers and blood disorders.

Introduction

Some of these treatments may cure your condition. Others will help relieve your symptoms on a day-to-day basis, and are called non-curative treatments.

There are lots of common questions around treatments. Here we provide some of the answers and link to websites where you can find more information.

What's the best treatment option for me?

When it comes to deciding on the best treatment, many factors need to be considered. These include:

  • the condition you have
  • how early it's been diagnosed
  • your symptoms and how likely they are to respond to treatment
  • genetics – a drug may be available that specifically targets the cause of your condition
  • your age and general health.

Some conditions can be managed through non-curative treatments such as regular blood transfusions – especially if you have a blood disorder. If you have a blood cancer you might be given chemotherapy or radiotherapy first, to stop abnormal cells growing and dividing. When they stop growing and dividing, this is called being in remission.

A stem cell transplant will be considered if:

  • treatments have been unsuccessful
  • your symptoms are becoming difficult to manage
  • there is a high risk of relapse.

Your medical team will be able to give you specifics about your situation, which will be personalised to you.

What is chemotherapy?

Chemotherapy uses anti-cancer drugs to destroy cancer cells. Cancerous or abnormal cells divide more quickly than normal cells, but chemotherapy drugs disrupt the way they grow and divide.

Unfortunately, these drugs also attack healthy cells. This is why many patients experience side effects like nausea, tiredness and hair loss during treatment. The healthy cells usually recover from chemotherapy. But cancer cells cannot recover.

Chemotherapy aims to get your condition into remission. Remission is where abnormal cells are no longer growing and dividing.

Doctors will often combine different chemotherapy drugs to create the best conditions for remission. They may also use other anti-cancer drugs and radiotherapy to do this.

Chemotherapy is also given in preparation for a stem cell transplant. It removes the cells of your immune system and makes space for the new stem cells you receive from your donor. This treatment is known as conditioning therapy.

More information on chemotherapy is available from Macmillan.

What is radiotherapy?

Radiotherapy works by using high energy X-rays to kill cancer cells. It’s an effective first line of treatment for some leukaemias and lymphomas.

Radiotherapy is delivered by specialists who can target the correct dose to a precise area of the body.

Unfortunately, radiotherapy also damages healthy cells. This can cause side effects which vary greatly between people. Some experience mild symptoms, such as tiredness. Others may have side effects that make them feel very unwell. These side effects normally go away within weeks of stopping treatment.

More information on radiotherapy is available from the NHS website.

As with chemotherapy, radiotherapy is used to prepare a patient for a stem cell transplant as part of the conditioning therapy. It will usually be given to your whole body. This is known as total body irradiation (TBI).

More information on TBI is available from Cancer Research UK.

What is CAR T-cell therapy?

CAR T-cell therapy modifies the immune system to help it fight cancer cells. 'CAR' stands for chimeric antigen receptor. Though it’s not right for everyone, it can be lifesaving for people who haven’t responded to other treatments.

T cells are a type of white blood cell and part of your immune system. They recognise and destroy abnormal cells, such as cancer cells. With CAR-T, scientists collect your T cells and change their DNA. This ‘trains’ them to fight your cancer. The modified CAR-T cells are then infused back into your body. You’ll need a course of chemotherapy first to create space for them.

CAR-T is a complex process. Specialists will need to agree it’s the right treatment for you. You’ll have lots of tests and spend about 28 days in hospital. Sometimes radiotherapy is needed. Side effects vary from person to person and can be serious.

More information on CAR-T is available in our web section What is CAR T-cell therapy?

What is gene therapy?

Gene therapy changes the genetic material in your cells to treat or cure a disease.

Gene therapy is currently only available to a few people with blood disorders. This includes some people who have:

Gene therapies are also available for some other genetic conditions that aren’t related to blood cancer or blood disorders.

Some gene therapies now use stem cells. These stem cells are autologous, which means they are the patient’s own stem cells.

If you are interested in whether gene therapy might be an option for you, please speak to your medical team. They will know your situation best and whether you’re eligible for gene therapy.

We have further information on gene therapy on our webpage Gene therapy and stem cells.

Information published: 04/04/2025

Next review due: 04/04/2028