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.

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2025
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