Comparison of efficacy and safety of thalidomide vs hydroxyurea in patients with Hb E-β thalassemia-a pilot study from a tertiary care Centre of India

Introduction: Hemoglobin (Hb)-F inducers are known to improve Hb level and transfusion dependence in thalassemia. This pilot study was conducted to assess the efficacy and safety of Hb-F inducer thalidomide compared to hydroxyurea (HU) in Hb E-beta thalassemia patients. Methods: This was a prospective interventional single-centre study with 45 Hb E-beta thalassemia patients equally divided into group-I (thalidomide+folic acid), group-II (HU + folic acid) and group-III (folic acid). Response was assessed at various time intervals with 12-months follow up period. Primary end points were increment in Hb, Hb-F level and improvement in transfusion requirement; secondary end point were tolerability and safety. Results: There was 100% responder (R: Hb-increment >1 g/dl) in group-I with 66.67% major responder (MaR: Hb-increment >2 g/dl), while there were 40% and 0% responder in group-II and III respectively. Hb-increment was significantly (p-value <0.0001) better in thalidomide arm compared to HU. The Hb-increment was attributable to both increase in Hb-F levels and reduction in ineffective erythropoiesis in thalidomide arm. Transfusion reduction was significantly better in group-I compared to group-II (100% vs 34%). No severe adverse effects was reported by patients of any group. Conclusion: Thalidomide showed a persistent significant Hb-increment and transfusion independence in Hb E-beta thalassemia patients compared to HU.

Automated summary

This study found that in patients with Hb E-β thalassemia, the use of thalidomide significantly increased Hb levels and Hb-F levels, reduced the need for transfusions, and had good tolerability compared to the use of hydroxyurea.