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Thalassaemia Treatments

My Thal

My Thal

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Thalassaemia usually requires lifelong treatment with blood transfusions and medication.

Children and adults with thalassaemia will be supported by a team of different healthcare professionals working together at a specialist thalassaemia centre.

Your healthcare team will help you learn more about thalassaemia and work with you to come up with an individual care plan that takes all your needs and health concerns into account.

Blood transfusions

Most people with thalassaemia major or other severe types will need to have regular blood transfusions to treat anaemia.

This involves being given blood through a tube inserted into a vein in your arm. It's usually done in hospital and takes a few hours each time.

How often you need to have transfusions depends on the type of thalassaemia you have.

People with the most severe type, beta thalassaemia major, may need a blood transfusion about once a month. Those with less severe types may only need them occasionally.

Blood transfusions are very safe, but they can cause too much iron to build up in the body, so you'll need to take medicine to remove the excess iron.

Medicine to remove excess iron

Treatment to remove excess iron caused by regular blood transfusions is known as chelation therapy.

It's very important because high levels of iron in the body can damage organs.

The treatment will usually need to start once you or your child has had around 10 blood transfusions.

Medicines used in chelation therapy are known as chelating agents.

There are 3 chelating agents currently available:

desferrioxamine (DFO) – given as an infusion, where a pump slowly feeds the liquid medicine through a needle into your skin over 8 to 12 hours; this is done 5 or 6 times a week
deferiprone (DFP) – taken as a tablet or liquid 3 times a day; it's sometimes used alongside DFO to reduce the number of infusions you need
deferasirox (DFX) – taken once a day as a tablet that you dissolve in a drink

Each medicine has its own advantages and disadvantages. Your healthcare team will help you decide which is likely to be best for you or your child.

Stem cell or bone marrow transplants

Stem cell or bone marrow transplants are the only cure for thalassaemia, but they're not done very often because of the significant risks involved.

Stem cells are produced in bone marrow, the spongy tissue found in the centre of some bones, and have the ability to develop into different types of blood cells.

For a stem cell transplant, stem cells from a healthy donor are given through a drip into a vein.

These cells then start to produce healthy red blood cells to replace the cells affected by thalassaemia.

A stem cell transplant is an intensive treatment that carries a number of risks.

The main risk is graft versus host disease, which is a life-threatening problem where the transplanted cells start to attack the other cells in your body.

For people with serious types of thalassaemia, the long-term benefits of a stem cell transplant will need to be considered against the possible risks to help determine whether the treatment is suitable.

Treating other problems

Thalassaemia can also cause a number of other health problems that may need to be treated.

For example:

hormone medication may be used to help trigger puberty in children with delayed puberty and treat low hormone levels
vaccinations and antibiotics may be recommended to prevent and treat infections
thyroid hormones may be used if there's a problem with your thyroid gland (hypothyroidism)
medicines called bisphosphonates may be used to help strengthen your bones
gallstones may be treated with gallbladder removal surgery

https://www.nhs.uk/conditions/thalassaemia/treatment/

Clinical Trials

New treatment trials in thalassaemia

Luspatercept

Gene Therapy

Gene Therapy

Blood transfusion

A blood transfusion is when you're given blood from someone else (a donor). It's a very safe procedure that can be lifesaving.

Why it's done

A blood transfusion may be needed if you have a shortage of red blood cells.

This may be because your body's not making enough red blood cells or because you have lost blood.

For example, you may need a blood transfusion if you have:

a condition that affects the way your red blood cells work – such as sickle cell disease or thalassaemia
a type of cancer or cancer treatment that can affect blood cells – including leukaemia, chemotherapy or stem cell transplants
severe bleeding – usually from surgery, childbirth or a serious accident

A blood transfusion can replace blood you have lost, or just replace the liquid or cells found in blood (such as red blood cells, plasma or cells called platelets).

Ask your doctor or nurse why they think you might need a transfusion if you're not sure.

What happens

Before having a blood transfusion, the procedure will be explained to you and you'll be asked to sign a consent form.

A sample of your blood will also be taken to check your blood group.

You'll only be given blood that's safe for someone with your blood group.

During a blood transfusion:

You sit or lie down in a chair or bed.
A needle is inserted into a vein in your arm or hand.
The needle is connected to a tube and a bag of blood.
The blood runs through the tube into your vein.

It can take up to 4 hours to receive 1 bag of blood, but it's usually quicker than this.

You can normally go home soon after, unless you're seriously unwell or need a lot of blood.

How you might feel during and after

You might feel a sharp prick when the needle is first inserted into your vein, but you should not feel anything during the transfusion.

You'll be checked regularly while receiving the blood. Tell a member of staff if you feel unwell or uncomfortable.

Some people develop a temperature, chills or a rash. This is usually treated with paracetamol or by slowing down the transfusion.

Your arm or hand may ache and have a bruise for a few days after.

Contact a GP if you feel unwell within 24 hours of having a blood transfusion, especially if you have difficulty breathing or pain in your chest or back.

Risks

Blood transfusions are common and very safe procedures.

All donor blood is checked before it's used to make sure it does not contain serious infections such as hepatitis or HIV.

There's a very small risk of complications, such as:

an allergic reaction to the donor blood
a problem with your heart, lungs or immune system (the body's defence against illness and infection)

The risks will be explained before having a transfusion, unless this is not possible – for example, if you need an emergency transfusion.

Speak to your doctor or nurse if you have any concerns.

Alternatives

A blood transfusion will only be recommended if it's needed and other treatments will not help.

If it's possible that you'll need a transfusion (for example, if you're due to have surgery or you have anaemia), you may sometimes be given medicine to:

lower your risk of bleeding, such as tranexamic acid
boost your number of red blood cells, such as iron tablets or injections

These can reduce your chances of needing a blood transfusion.

Giving blood afterwards

Currently, you cannot give blood if you have had a blood transfusion.

This is a precautionary measure to reduce the risk of a serious condition called variant CJD (vCJD) being passed on by donors.

Find out more about who can give blood on the NHS Blood and Transplant website.

Information:

The NHS Blood and Transplant website has a range of patient information leaflets including:

information for parents and children
blood transfusion in pregnancy
alternatives to blood transfusion

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