Efficacy of Thalidomide in Elimination/Reduction of Blood Transfusions and Iron Chelation

The optimal management of β-thalassemia includes regular blood transfusion and iron chelation. The availability of blood for this purpose is limited and this was acutely felt during Covid. The medical community has been striving for various methods to minimise/eliminate the necessity of blood transfusions in the management of thalassemia. "Luspatercep" is one such option, but it is not an affordable option for most Indians. The other probable alternative is a low dose of thalidomide under medical supervision. A few studies have been carried out both within and outside India to prove its efficacy. Unfortunately, these studies were carried out on a small number of patients and for a limited period. In 2022, Pahuja Trust sponsored a two-year trial in which nearly 150 patients from five different hospitals in the country participated. The interim report of the Study shows promising results – the report is included on this website. The study shall be completed by August 2024. Hopefully, the authorities will approve the recommendations of the Study which will effectively eliminate/minimise the frequency of blood transfusion and the use of iron chelation.

There are nearly 10,000 children with thalassemia major who are born in India every year. One of the methods to prevent their births is to screen the expectant mothers to confirm if they are carriers and if so, thereafter screen the father. The Trust had planned to launch a pilot project in Delhi during 2023 but due to some unforeseen reasons, it could not be done. An effort is being made to do so in Dehradun in mid-2024.