اثر و عوارض جانبي هيدروكسي اوره در بيماران تالاسمي اينترمديا

Efficacy and side effects of hydroxyurea in patients with thaIasemia intermedia

Introduction: The ~-thalas saemias represent a heterogenous gruup of diseases resulting from decreased p-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 halance in globin chain synthesis. Hydroxyurea (HU), as an effective with low toxicity for activating y-globingene, has beenshuwn10 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 starred their regular blood transfusion beyond of 2 years of age and werebeing treated with lD-12 mglkg/d HU orally, Results: The study group consist of 38 men and 42 women. The average age was l6 .8(±6.9) years and thalassemia was diagnosed according to the RBCindexes 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 wereseen 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 .