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<p>E beta (EB) thalassemia is a common hemoglobinopathy in eastern India with heterogeneous clinical presentation that ranges from asymptomatic disease to transfusion dependent thalassemia (TDT). Thalidomide is an immunomodulator which increases HbF level and mitigates the imbalance between alpha and globin chains and therefore modifies the clinical outcome of the disease. This single-arm arm, prospective, open-label study enrolled patients with EB Thalassemia between 12 and 65 years, who are transfusion-dependent and refractory to hydroxyurea. Subjects were initiated on thalidomide at 50 mg for 52 weeks. The primary objective was to assess the reduction in transfusion requirement at the end of 52 weeks. The secondary objectives were to assess the increment in hemoglobin (Hb) level, reduction in ferritin and toxicity. 49 patients were included in final analysis. The mean age was 23 years. Transfusion requirement at the end of 52 weeks when compared to baseline was 4.1 ±6.6 units/year vs 18.1 ± 5.6 units/year (<i>p</i> &lt; 0.001). Overall response was noted in 90% patients. Complete response was seen in 69%, partial response in 21% and 10% patients had no response. Mean hemoglobin pre-treatment and post treatment was 6.9 ± 0.8 g/dl and 8.0 ± 0.2 g/dl respectively (<i>p</i> &lt; 0.005). Mean ferritin level pre-treatment and post-treatment was 2,571.5 (± 1894.9) ng/ml and 2,017.7 (± 1,156.2) ng/ml respectively (p&lt;0.001). Most common toxicities were constipation and sedation. No grade III/IV toxicity noted. Thalidomide is effective at low dose and safe in patients of TDT E beta Thalassemia.</p>

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Efficacy and Safety of Low Dose Thalidomide in Transfusion-Dependent E-beta (EB) Thalassemia Refractory to Hydroxyurea- A Single Arm Prospective Analysis

  • 期刊论文

  • 发表日期：

    2026年5月2日

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  • Dibakar Podder,
  • Pampi Majumder,
  • Maitreyee Bhattacharya

摘要

E beta (EB) thalassemia is a common hemoglobinopathy in eastern India with heterogeneous clinical presentation that ranges from asymptomatic disease to transfusion dependent thalassemia (TDT). Thalidomide is an immunomodulator which increases HbF level and mitigates the imbalance between alpha and globin chains and therefore modifies the clinical outcome of the disease. This single-arm arm, prospective, open-label study enrolled patients with EB Thalassemia between 12 and 65 years, who are transfusion-dependent and refractory to hydroxyurea. Subjects were initiated on thalidomide at 50 mg for 52 weeks. The primary objective was to assess the reduction in transfusion requirement at the end of 52 weeks. The secondary objectives were to assess the increment in hemoglobin (Hb) level, reduction in ferritin and toxicity. 49 patients were included in final analysis. The mean age was 23 years. Transfusion requirement at the end of 52 weeks when compared to baseline was 4.1 ±6.6 units/year vs 18.1 ± 5.6 units/year (p < 0.001). Overall response was noted in 90% patients. Complete response was seen in 69%, partial response in 21% and 10% patients had no response. Mean hemoglobin pre-treatment and post treatment was 6.9 ± 0.8 g/dl and 8.0 ± 0.2 g/dl respectively (p < 0.005). Mean ferritin level pre-treatment and post-treatment was 2,571.5 (± 1894.9) ng/ml and 2,017.7 (± 1,156.2) ng/ml respectively (p<0.001). Most common toxicities were constipation and sedation. No grade III/IV toxicity noted. Thalidomide is effective at low dose and safe in patients of TDT E beta Thalassemia.