A prospective study to understand the clinical efficacy, safety, and molecular mechanisms of thalidomide-induced erythropoiesis in patients with non-transfusion dependent thalassemia (NTDT)

**Introduction:**Non-transfusion dependent thalassemia [NTDT] is a milder form of thalassemia in which patients require transfusions every 3-4 months. Hydroxyurea shows improvement in 30-40% while thalidomide used as second line treatment has shown improvement in 50%.

Methodology: We would like to do perform a Phase II randomized controlled trial in which newly diagnosed patients with NTDT will be randomized to receive either Hydroxyurea or Thalidomide for a period of 12 months. At the end of 12 months, patients will be classified as responders or non-responders. Patients will also have samples collected to identify genetic markers that predict response to thalidomide  as well as study mechanisms of thalidomide induced erythropoiesis.

**Results:**We will try to study if thalidomide is a better drug than hydroxyurea for reducing transfusion frequency in NTDT. In the same study, we will try and identify predictors of response to thalidomide.

Conclusions: We hope to show improved efficacy of thalidomide in patients with NTDT with a reduction in transfusion frequency. We also hope to elucidate mechanisms for response to thalidomide in patients with NTDT.