Thalidomide has a significant effect in patients with thalassemia intermedia.

Objective: To investigate the effect of thalidomide in patients with thalassemia intermedia. Methods: We observed the effect of thalidomide in seven patients with thalassemia intermedia requiring blood transfusion. Four of the patients were transfusion-independent, and three patients were transfusion-dependent. Results: For the four transfusion-independent patients, hemoglobin concentration increased significantly (>= 2 g/dl) in three and moderately ( 1-2 g/dl) in one. After 3 months of treatment, hemoglobin concentration increased 3.2 +/- 1.2 g/dl compared to pretreatment. Among the three transfusion-dependent patients, transfusion was terminated after one month of treatment in one patient and decreased > 50% in the other two patients, accompanied by an increase in the average hemoglobin concentration. Conclusion: Thalidomide had a significant effect in patients with thalassemia intermedia. Further studies of a larger scale and more rigorous design are warranted.