General Updates | Past studies that have assessed the safety and efficacy of thalidomide in thalassemia

So, over the last few years, I think since, you know, about 2015-16 onwards, there have been many studies which have been published, but these studies mainly come from, you know, four countries in the low-middle-income countries in Southeast Asia, that’s India, Pakistan, some from Bangladesh, Iran, and a large body of evidence comes from China, right? So these are four or five countries from where all the literature about thalidomide is being published. There are multiple papers, a lot of them are actually just prospective single-arm trials, there is no, except for two studies, there is no head-to-head comparison with you know a placebo or the standard of care treatment like your regular transfusion chelation. And you know the population that has been studied in these trials have been very variable – some of the studies had only transfusion-dependent, the others had a combination of transfusion-dependent and non-transfusion-dependent thalassemia. The age group is very varying, the endpoints, the response criteria have been very different, so it’s very difficult to put all of this together, but most studies have shown that it helps in improving hemoglobin by actually increasing HbF, because you know the increase in HbF correlated with or was proportional to the increase in HbF. 

A lot of studies are mainly you know concentrated on adults, but few studies from Pakistan and from India have looked at children, which remains a concern right now because of safety measures. So considering you know there are more than 20 papers right now which talk of thalidomide and thalassemia and they’ve shown that it increases hemoglobin, it reduces transfusion requirement, but safety is something which I believe you know none of these papers have looked at it the way it should have been, considering our past experience or not so good experience with thalidomide in the past, right? And that’s why the drug was not being used for so many years. And so yes, it is efficacious, I agree with that, but safety is something that we need to look at, especially in thalassemia, because it’s not going to be a short-term use, it’s not something that you’re going to use for one year or two years, it’s going to be used lifelong, so long-term safety remains a concern, and especially if we’re using it in a very young child. So we need to have certain parameters or certain long-term studies before we can call it safe for use in thalassemia and more so in pediatric patients with thalassemia.

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