Treatment for beta-thalassemia depends heavily on severity, ranging from no treatment at all for most carriers to intensive, lifelong care for beta-thalassemia major.

• Regular blood transfusions. For beta-thalassemia major, transfusions every two to five weeks are the foundation of treatment, keeping haemoglobin high enough to support normal growth and development and to suppress the overactive, damaging bone marrow response described earlier. Some individuals with beta-thalassemia intermedia also need transfusions occasionally, such as during illness, surgery, or pregnancy.
• Iron chelation therapy. Regular transfusions, and in intermedia even increased absorption of iron from food, cause iron to build up in the heart, liver, and hormone-producing glands over years, which can be life-threatening if untreated. Medications called iron chelators bind this excess iron so the body can remove it, and are essential alongside transfusion therapy. Options include an injectable medication, deferoxamine, and two oral medications, deferiprone and deferasirox, chosen based on individual response, organ involvement, and tolerability.
• Luspatercept. This newer injectable medication helps red blood cells mature more effectively in the bone marrow, and has been shown to meaningfully reduce transfusion needs in some individuals with transfusion-dependent beta-thalassemia.
• Mitapivat. An oral medication that activates an enzyme called pyruvate kinase to support healthier red blood cell function, studied both in individuals who need regular transfusions and those who do not.
• Folic acid supplementation. Commonly given to support the bone marrow’s increased demand for building blocks to make red blood cells.
• Splenectomy, or surgical removal of the spleen, may be considered when the spleen enlarges significantly and is driving up transfusion requirements, though it is generally delayed as long as possible because it raises long-term infection risk.
• Hematopoietic stem cell transplantation. This remains the only established cure for beta-thalassemia, replacing the affected bone marrow with healthy stem cells, usually from a matched sibling donor. Outcomes are best when performed at a younger age, before iron overload and organ damage have accumulated, and several centres in India have experience performing this transplant for beta-thalassemia.
• Gene therapy. The newest treatment approach modifies a person’s own stem cells, either by adding a working copy of a beta-globin gene or by switching on the body’s fetal form of haemoglobin, so that transfusions are no longer needed. Two such therapies, betibeglogene autotemcel and exagamglogene autotemcel, have shown that the large majority of treated individuals no longer need regular transfusions afterward, and are approved in some countries, though availability, cost, and infrastructure currently limit access to this treatment in India.

Care for beta-thalassemia major or intermedia is generally best coordinated through a haematologist experienced in thalassemia, working alongside specialists for the heart, hormone system, and bones as needed, since the condition and its treatments affect multiple organ systems over a lifetime.