Safety and Efficacy of Thalidomide and Hydroxyurea Combination in Beta Thalassemia Patients

Beta thalassemia results from imbalance in alpha and beta globin chains causing severe anemia, transfusion dependency, and iron overload. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment. Patients without the option of HSCT may benefit from Hemoglobin F (HbF) inducing agents like thalidomide and hydroxyurea (HU). We conducted a retrospective analysis on 87 beta thalassemia patients who received a combination of low dose thalidomide and HU from January 2017 to December 2020. Patients received combination of HU 500 mg everyday (> 30 kg) or every alternate day (< 30 kg) and thalidomide 100 mg (> 30 kg) or 50 mg (< 30 kg) once daily. Parameters such as transfusion requirement, anthropometry, Hb levels, ferritin, drug side effects etc. were monitored and evaluated at the end of one year of therapy. Sixty-three patients (72%) achieved transfusion independence and were eligible for the study. Median time to transfusion independence was 6 months (range 3-11 months). At the end of 1 year, overall response rate was 72%. There was significant improvement in the Hb levels, ferritin values and height at the end of 1 year of follow up. No grade 3 or 4 toxicities were noted. We document improvement of Hb levels, transfusion independence, reduction in iron overload, and improvement in growth parameters with minimal side effects at the end of 1 year of follow up.