Treatment

Thalassaemia usually requires lifelong treatment with blood transfusions and medicine.

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 and support 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.

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 swallow or 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 medicine 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

You may also need regular monitoring to check for heart problems.