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General Updates | The mechanism of action and rationale for using thalidomide in thalassemia
Shruti Kakkar, MD, Christian Medical College & Hospital, Ludhiana, India, discusses the rationale for thalidomide as a potential therapeutic option in patients with thalassemia, highlighting its immunomodulatory and anti-angiogenic properties. Dr Kakkar notes that thalidomide improves the symptoms of thalassemia through several mechanisms, with the primary mechanism being increased fetal hemoglobin (HbF). This interview took place virtually.
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Transcript
So thalidomide, as you know, all of us know, it is known as an immunomodulatory and an anti-angiogenic agent. The effect of thalidomide was probably found out, you know, by chance in a patient who had thalassemia, along with that he developed multiple myeloma long back in 2004. And when thalidomide was given for treating multiple myeloma the Hb also improved and then over a period of time there have been studies there have been, you know, animal models which have shown that how thalidomide increases the gamma globin mRNA leading to increased HbF...
So thalidomide, as you know, all of us know, it is known as an immunomodulatory and an anti-angiogenic agent. The effect of thalidomide was probably found out, you know, by chance in a patient who had thalassemia, along with that he developed multiple myeloma long back in 2004. And when thalidomide was given for treating multiple myeloma the Hb also improved and then over a period of time there have been studies there have been, you know, animal models which have shown that how thalidomide increases the gamma globin mRNA leading to increased HbF. That increase in HbF results in improvement in hemoglobin in patients with both transfusion-dependent and non-transfusion-dependent thalassemia.
So this is one of the effects but there are other effects as well, like you know, being an anti-angiogenic agent. It helps reduce the splenic congestion and helps with hypersplenism. Patients with thal who have gone into hypersplenism, so it helps in that situation, It reduces extramedullary hematopoiesis. And over a period of time, if the transfusion requirement comes down, then probably we hope it will act on iron overload as well and result, and you know, finally result in reduced iron overload.
One very interesting study that I found recently was how it, you know, reduces the endothelial stress in patients with thalassemia, especially those who were planned to undergo a transplant. So reducing endothelial stress meant less chance of veno-occlusive disease. So that could be one, you know, action that we may, you know, look at it in the future, so that our patients with thalassemia who had high iron overload and are at high risk of veno-occlusive disease, if that can be reduced by bridging therapy with thalidomide while going for transplant, so it’s definitely multi… the mechanism is not absolutely known, there are multiple points at which thalidomide is acting, but definitely we see an increase in HbF that helps improve all the symptoms in patients with thalassemia.
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