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Treating beta thalassaemia

The best way to manage beta thalassaemia depends on various factors.

Your healthcare team will work with you to find the most suitable treatment for you, considering your preferences and requirements. Let’s look at some of the therapies they may prescribe.

Gaia living with beta thalassaemia talking with someone

Gaia, living with beta thalassaemia major in Italy

    People with transfusion-dependent beta thalassaemia will have regular transfusions, while those with non-transfusion-dependent beta thalassaemia may only occasionally need transfusions.1

    Beta thalassaemia reduces your body’s ability to make haemoglobin, the protein in red blood cells that carry oxygen from the lungs to the rest of the body.2,3 It also causes red blood cells to die early.1,3

    That’s why people living with beta thalassaemia have fewer red blood cells than normal (anaemia), which can make a person feel tired.3,4 To find out more about how beta thalassaemia affects your red blood cells, click here.

    Blood transfusions can help with the symptoms of anaemia by providing your body with healthy red blood cells.1 They also aim to ensure the growing body is developing normally and that your energy levels are good.2

    Blood transfusion means receiving someone else’s blood through a tube placed into a vein in your arm.5 The blood comes from blood donors and undergoes careful checks before you get it.2,6

    Whether and how often you need blood transfusions depends on the type of beta thalassaemia you have:

    • Transfusion-dependent beta thalassaemia requires receiving transfusions regularly, typically once a month.1
    • People living with non-transfusion-dependent beta thalassaemia may occasionally need a transfusion (for example due to a lack of growth or during an infection), but they rarely need regular transfusions. Transfusions may also be required when the need for red blood cells increases, for instance, during periods of stress.1,4

    Want to know more about the different types of beta thalassaemia? You’ll find more information here.

    Receiving regular transfusions means spending a lot of time at the hospital. That can be hard to fit into a busy life. However, getting your transfusions when you need them is essential to ensure your condition doesn’t get worse.6 Stay in contact with your healthcare team and let them know if there are ways to make it easier for you to stick to your treatment.

      Iron overload

      People with beta thalassaemia may have too much iron in their bodies (called “iron overload”), which has different causes.

      Regular blood transfusions are the most common cause of iron overload in transfusion-dependent beta thalassaemia.1,2 People living with non-transfusion-dependent beta thalassaemia may also have iron overload, even though they don’t have regular transfusions, but this often does not happen until adulthood.1,2
       

      Iron chelation therapy

      Too much iron is a problem because your body can’t get rid of it.1,2 So, the excess iron gathers in organs like your heart, liver, and endocrine system, where it can cause damage.

      To avoid that, your healthcare team may prescribe a medication called iron chelation therapy.1

      There are different types of iron chelation drugs, some that you take orally and others that you receive through an injection or an infusion in your vein. Your healthcare team will speak to you about the options which may best suit you.1

        A stem cell transplant is currently the only curative treatment option that can provide long-term relief from the symptoms and burden of transfusions in transfusion-dependent beta thalassaemia.1-3

        Although stem cell transplantation is beneficial to the patient as a potential cure, a lack of appropriate stem cell donors means it is not an option for most patients.2 Your healthcare team will decide if the transplant route is a suitable and available option for you.

        A stem cell transplant involves the person with beta thalassaemia receiving stem cells from a suitable donor.3 These stem cells have the potential to develop into healthy new red blood cells.3

        The person getting the transplant will undergo several treatments, which may include chemotherapy or radiotherapy, before the transplant to prepare the body for receiving the foreign stem cells. This preparation is called ’conditioning’. Likewise, they will need to continue taking some medicines for some time after the procedure to ensure that the body doesn’t reject the transplant.3

        Some of the pre-transplant ‘conditioning’ steps can cause side effects, including effects on fertility.7 If you think you may want to have children (even if not now but at some time in the future) then talk with your healthcare team about your options.

        For example, eggs, embryos or sperm can be frozen before you have a transplant for you to use if you want to start a family at a later date, and other options may be available to you.7 Depending on where you live, funding and support for fertility and in vitro fertilisation (IVF) services may be available from your local trust.

        Naziha's hands

        Everyone is different

        There’s no single way to treat beta thalassaemia. The optimal care for you depends on your symptoms and needs. If you have any questions about managing your beta thalassaemia, keep talking with your healthcare team.

        The treatments mentioned in this article are some of the ones your doctor may mention. This is not a complete list.

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        Resource Hub

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          1. National Health Service (NHS). Thalassaemia: Treatment. 2022. https://www.nhs.uk/conditions/thalassaemia/treatment/ Accessed 20 May, 2023.
          2. Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010;5:11. doi:10.1186/1750-1172-5-11.
          3. Cappellini MD, Farmakis D, Porter J, Taher A. 2021 Guidelines for the management of transfusion dependent thalassaemia (TDT). 4th Edition. IDF, 2021.
          4. Needs T, Gonzalez-Mosquera LF, Lynch DT. Beta thalassemia. 2022. https://www.ncbi.nlm.nih.gov/books/NBK531481/ Accessed 20 May, 2023.
          5. Taher AT, Radwan A, Viprakasit V. When to consider transfusion therapy for patients with non-transfusion-dependent thalassaemia. Vox Sang.2016; 108:(1):1–10. doi: 10.1111/vox.12201.
          6. National Institute of Health (NIH). Treatments for blood disorders. 2022. https://www.nhlbi.nih.gov/health/blood-bone-marrow-treatments. Accessed 20 May, 2023.
          7. Aserlind A, Martini A, Dong J, et al. Fertility preservation before hematopoetic stem cell transplantation: a case series of women with GATA binding protein 2 deficiency, dedicator of cytokinesis 8 deficiency, and sickle cell disease. F S Rep. 2020;1(3):287–293. doi: 10.1016/j.xfre.2020.10.001.