Thalassemia can be well managed with the right treatment — and the right treatment depends on which type a person has and how severe it is. In severe thalassemia the body has fewer red blood cells and less hemoglobin, so the anemia is worse.
Who needs transfusions
A blood transfusion — a safe, common procedure that delivers blood through a small plastic tube into a blood vessel — gives the body more red blood cells to carry oxygen. It is the main treatment for moderate and severe thalassemia.
| Type | Transfusions |
|---|---|
| Thalassemia major | often needed regularly, because the body makes so little hemoglobin |
| Thalassemia intermedia — milder than major, more serious than trait | sometimes, for example during an infection or illness |
| Thalassemia minor or trait | usually not needed: there is no anemia, or only mild anemia |
Folic acid, a B vitamin that helps red blood cells develop, is often prescribed alongside other treatments.
The costs of frequent transfusions
Iron overload
Red blood cells are rich in iron. Over many transfusions, iron builds up in the body and collects in the heart, liver and brain, where it can stop them working properly. Chelation therapy — a medicine taken as a pill or as a shot under the skin — removes the excess iron before it builds up in the organs. People who are transfused regularly may need it on an ongoing basis.
Alloimmunization
Each transfusion raises the risk that the immune system will treat donated blood as harmful and try to destroy it. People with alloimmunization can still be transfused, but their blood has to be carefully matched first — which takes time, and can mean a longer wait or more trouble finding suitable blood.
Infection
Some infections, such as hepatitis, can be carried in blood. The U.S. blood supply is screened and monitored, so the risk of infection from a transfusion is very low — but not zero. CDC has more on transfusion complications in thalassemia.
Gene therapy
In January 2024, the U.S. Food and Drug Administration approved Casgevy (exagamglogene autotemcel, or exa-cel), a cell-based gene therapy for transfusion-dependent beta-thalassemia in patients aged 12 and older.
- The patient's own blood stem cells are collected.
- The cells are modified in the lab.
- The patient has high-dose chemotherapy, which clears cells from the bone marrow to make room.
- The modified cells are given back in a one-time infusion, as part of a blood stem cell transplant.
More is on the FDA website and from the Cooley's Anemia Foundation.
CDC has also published videos in which a man living with thalassemia talks about moving to adult care, how iron overload affects the body, and why sticking with treatment matters.
Sources
Based on "Treatment of Thalassemia," Centers for Disease Control and Prevention; a work of the United States government in the public domain. A stock photograph is left out.
ライセンス： CC0 1.0（パブリックドメイン） · 出典 www.cdc.gov
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