What is it about?

We followed 164 patients with transfusion-dependent beta-thalassaemia for 30 months: 72 received thalidomide and 92 continued standard transfusions. Haemoglobin rose substantially in the thalidomide group, and response was linked with GATA-1 and KLF gene expression, age at first transfusion and an HBS1L-MYB genetic variant.

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Why is it important?

Regular transfusions cause iron overload and other complications. These findings support further evaluation of thalidomide as a lower-cost treatment and suggest biological markers that may help identify patients most likely to benefit.

Perspectives

From my perspective, this work matters because Regular transfusions cause iron overload and other complications. I hope the findings support further research and informed, evidence-based decisions.

Yasar Yousafzai
Khyber Medical University

Read the Original

This page is a summary of: Thalidomide confers therapeutic benefit in beta thalassemia patients by enhancing hemoglobin and hematopoietic gene expression: A non-randomized clinical trial, Blood Cells Molecules and Diseases, July 2025, Elsevier,
DOI: 10.1016/j.bcmd.2025.102936.
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