Thalidomide and hydroxyurea combination therapy in transfusion-dependent beta-thalassemia major: A real-world retrospective analysis

Abstract Thalidomide and Hydroxyurea Combination Therapy in Transfusion-Dependent Beta-Thalassemia Major: A Real-World Retrospective Analysis Author: Shyam Rathi, MD, DM Hematology Superspeciality Center, Mumbai Context: Transfusion-dependent beta-thalassemia major poses significant challenges in developing countries due to limited access to curative therapies like bone marrow transplantation. Objective: To evaluate the efficacy and safety of thalidomide and hydroxyurea combination therapy in patients with transfusion-dependent beta-thalassemia major. Methods: We conducted a retrospective analysis of 18 patients with transfusion-dependent beta-thalassemia major who received thalidomide and hydroxyurea combination therapy. The median age was 12 years (range 3-24), with 11 males and 7 females. Results: The combination therapy resulted in a good response in 11 patients (61%) with significant improvement in hemoglobin levels (>9 g/dL), while 4 patients (22%) showed a partial response. The therapy was well-tolerated, with manageable side effects. Conclusion: Thalidomide and hydroxyurea combination therapy may offer a viable treatment option for transfusion-dependent beta-thalassemia major, particularly in resource-constrained settings. Further studies are needed to confirm these findings and explore long-term outcomes. Support: None Conflict of Interest Disclosure: The author declares no conflicts of interest. Off-label Disclosure: Thalidomide is used off-label for the treatment of transfusion-dependent beta-thalassemia major.