Thalassaemia

Treatment

Thalassaemia usually requires lifelong treatment with blood transfusions and medication.

The main treatmentsthat may beusedareoutlined below:

Children and adults with the condition will be supported by ateam of different healthcare professionals working together in a specialist thalassaemia centre.

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

Blood transfusions

Most people with thalassaemia major or other severe types will need to have regular Blood transfusion 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, butthose with less severe types may onlyneed themoccasionally.

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

Removing 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 tostart once you or your child has had around 10 blood transfusions.

Medications used in chelation therapy are known as chelating agents. There are three chelating agents currently available:

desferrioxamine (DFO)takenusing a pump that slowly feeds the medicine through a needle into theskin (infusion) over8-12 hours, five or six times a week
deferiprone (DFP)taken as atablet or liquid three times a day; it'ssometimes usedalongside 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 medication has itsown advantages and disadvantages. Your care 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, butthey're not done very often because of the significant risks involved.

Stem cells areproducedin 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 bythalassaemia.

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 ofother problems that may need to be managed. For example:

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