This is the eighth part of our series about the condition based on our patient booklet “Fast Facts for Patients: Alpha Thalassemia”. This article shows what clinical trials are and which new treatments are on the horizon.

Clinical Trials

If you are interested in new treatments, you may want to ask your doctor about clinical trials. All new medical treatments have to be tested in clinical trials. A new treatment must go through several phases of testing before it can be proven to work better than existing treatment and be adopted into routine care. A potential treatment will only move on to the next phase of research if it is safe and shows promise.

Clinical trials: preclinical, phase I, phase II, phase III

New Treatments for Alpha Thalassemia

Slowing the Breakdown of Red Blood Cells

There has been some research looking into treating anemia in people with AT with medication to reduce the destruction of red blood cells.

Mitapivat is a new treatment that is being tested in people with AT or beta thalassemia. It’s a tablet that you take twice a day. It’s already used to treat another genetic condition that causes anemia, called pyruvate kinase deficiency.

This treatment helps to activate an enzyme that is needed for red blood cells to function properly. Early trial results show that it may be able to reduce anemia in people with AT who don’t need regular blood transfusions. Side effects found so far include difficulty sleeping, headache and dizziness.

Mitapivat is being tested in a Phase III trial for people with AT who do and do not require regular transfusions.

Stem Cell Transplant

The only way to potentially cure AT is with a stem cell transplant from a donor. Stem cell transplant is currently only suitable for AT major, as the treatment itself has many side effects, some of which can be life-threatening.

Stem cells are cells in the bone marrow that can develop into all the different types of blood cell in the body, including red blood cells.

When you have a transplant, the stem cells in your bone marrow are destroyed and replaced with healthy cells from the donor. The donor must be someone whose blood cells closely match yours, and this is usually a close family member.

Alpha thalassemia: stem cell transplant (donor / patient)

The aim is for the donor stem cells to start to grow inside your bones and provide new blood stem cells to replace yours. This process is called ‘engraftment’. The new stem cells will produce all the different types of blood cells, including healthy red blood cells.

Until the new stem cells have started to work, you have a very high risk of infection. So, you need to be nursed in isolation for some time after having your stem cell infusion.

 

Please check out the other posts of our series here:

 

Information based on Fast Facts for Patients: Alpha Thalassemia (Karger, 2023).

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