Efficacy and side effects of hydroxyurea in patients with thalasemia

Introduction: The β-thalassaemias represent a heterogenous group of diseases resulting from decreased β-globin mRNA expression and imbalanced alpha/betaglobin chain synthesis which are clinically manifested by ineffective erythropoiesis and excessive hemolysis. Increasing levels of hemoglobin F (HbF) by improving the balance in globin chain synthesis. Hydroxyurea (HU), as an effective with low toxicity for activating y-globin gene, has been shown to enhance HbF synthesis. Methods: To analyze the clinical presentation and possible side effects of HU treatment 80 thalassemia intermedia patients were selected and followed-up for one year regularly. All of them started their regular blood transfusion beyond of 2 years of age and were being treated with 10-12 mg/kg/d HU orally. Results: The study group consist of 38 men and 42 women. The average age was 16.8(±6.9) years and thalassemia was diagnosed according to the RBC indexes and Hb electrophoresis. An improvement of erythropoiesis was reflected by an increase in hemoglobin concentration, the state of energy, fatigability, and mood. About 83% (69) of the patients tolerated the HU well and showed a dramatic response to the drug. Thirty of 80 transfusion-dependent patients became completely transfusion-free and 23 cases had one or two transfusions throughout the study. Vomiting and thrombocytopenia were seen in two patients. Conclusion: During this close observation, no malignant change was seen in our patients. HU therapy appears to be safe and effective when administered in thalassemic patients. Key words: Thalassemia