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What is the treatment of thalassaemia major?

Q: My 7 years old son is having thalassaemia major. How it can be treated? Please advise.

A:As you must be aware, Thalassaemia major results due to a genetic defect causing reduced rate of synthesis of one of the globin chains (usually when one says Thalassaemia Major one is talking about decreased synthesis of beta globin chain) that make up haemoglobin. Reduced synthesis of one of the globin chains can cause the formation of abnormal haemoglobin molecules, thus causing anaemia, the characteristic-presenting symptom of the thalassaemia. Treatment for patients with thalassemia major includes chronic blood transfusion therapy, iron chelation, and definitive curative option in allogeneic bone marrow transplantation (BMT). While there have been many advances in the chelation agents for reducing iron overload, the option of cure is only by BMT and that too with HLA matched allogeneic bone marrow. Cord blood transplantation, has also shown promising results, however, its use is limited.

I would suggest that you get in touch with a clinical haematologist & bone marrow transplantation physician for example in CMC, Vellore, BL Kapur Memorial Hospital, or AIIMS in New Delhi, etc. to guide you further.


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