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General Updates | The potential role of thalidomide in the treatment of thalassemia
Shruti Kakkar, MD, Christian Medical College & Hospital, Ludhiana, India, discusses the potential role of thalidomide in the treatment of thalassemia, highlighting its potential as an alternative for patients in low- to middle-income countries with limited access to other therapies. Dr Kakkar notes that thalidomide may be of value in situations where transfusion or effective iron chelation is not feasible. This interview took place virtually.
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Transcript
I think the effect that we’re seeing with thalidomide is quite interesting. And as I said earlier, I think safety is the major concern. If we can in future prove that it is safe, then probably it will be the best alternative for children in especially low- to middle-income countries where we still do not have access to drugs like luspatercept and mitapivat so yes but right now thalidomide should be restricted to certain situations if you’re not able to transfuse a patient because of probably alloimmunization or the requirement is so high like you know if a patient has developed hypersplenism and the annual transfusion rate is so high that you’re not able to chelate the patient well probably that’s one reason because thalidomide has shown its effect in reducing the spleen size right...
I think the effect that we’re seeing with thalidomide is quite interesting. And as I said earlier, I think safety is the major concern. If we can in future prove that it is safe, then probably it will be the best alternative for children in especially low- to middle-income countries where we still do not have access to drugs like luspatercept and mitapivat so yes but right now thalidomide should be restricted to certain situations if you’re not able to transfuse a patient because of probably alloimmunization or the requirement is so high like you know if a patient has developed hypersplenism and the annual transfusion rate is so high that you’re not able to chelate the patient well probably that’s one reason because thalidomide has shown its effect in reducing the spleen size right. If you know your patient is you do not have access to regular blood transfusions there is a shortage of blood and most patients are not transfusable that’s something that we would look at. And one or you know particular scenario would be lack of availability of good chelation. A lot of drugs in low-middle-income countries are generic drugs, and, you know, we come across patients who have severe cardiac and hepatic iron overload, and deferoxamine is not available or it’s not accessible. Then probably that’s one set of patients where you would like to, you know, add thalidomide and see if you can come down on you know transfusions and in future you know as the body utilizes that iron for erythropoiesis then that iron load may come down. So if you’re not able to transfuse you do not have access to safe blood or adequacy of blood cannot be ensured and if you cannot chelate your patient well, I think I would stick to that right now, but if you have good quality blood products chelators available then it’s stick to the standard of care regular transfusion chelation – that’s the standard of care as of today.
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