As you saw, the efficacy of thalidomide for raising the hemoglobin of thalassemics is proven. Dr Ramanan has implemented research done by others in his therapy, and has had much success using thalidomide with patients. Dr Ramanan made a judgment that took into account the cost of treatment, problems with compliance and blood safety in India and decided to try what had already been proven. It would be accurate to say that his patients are the test group, even though it is not a trial, but with no one willing to run trials, mostly due to the previous stigma associated with the use of higher doses of thalidomide in pregnant women in the 1960's, which caused birth defects in children, the doctor felt he had no choice. With the doses being used in thalassemia, there have not been any alarming side effects noted. Some patients have stopped transfusing now for several years. I think when a doctor like Dr Ramanan sees so many patients dying on a regular basis, utilizing an alternative approach to treatment would have to seem attractive, and so far it is working out.
I can see this as a viable alternative in countries where costs and blood safety are problems. India still has ongoing problems with blood safety. Since there is real hope for a new drug that will raise Hb significantly, I see thalidomide as a short term answer for some patients. Hopefully, we will see Luspatercept fulfill its promise and drugs like hydroxyurea and thalidomide will probably become outdated for thalassemia.
*Pregnant women should never take thalidomide.