hydroxyurea can reduce or eliminate transfusion requirements in children with major and intermediate thalassemia

background: hydroxyurea (hu) is a well known chemotherapeutic agent that has been used largely for various myeloproliferative diseases over the past 20 years. in β-thalassemia, the effect of hu is much less clear and remains controversial. this study was undertaken to describe the hematologic and clinical responses of thalassemia major and intermediate patients to hu treatment during 2 years. materials and methods: seventy one major and twenty transfusion-dependent intermediate thalassemia participants were selected among 150 β-thalassemia patients. all patients underwent laboratory tests, and the state of energy, social activity, tolerance, and mood were recorded in the beginning of the study. echocardiography was carried out before and during treatment with hu. all patients were treated with hu the initial dose was 10- 15mg/kg/day given once a day. results: all the patients tolerated hu well and showed a dramatic response to the drug. nine of 20 intermediate and 8 of 71 major patients became completely transfusion free. in 6 intermediate and 15 major patients, transfusion interval prolonged more than 50%. after treatment, 95% of intermediate and 81% of major patients described an increase in social activity. hu therapy was also associated with a marked decrease in serum ferritin level in major thalassemia patients. conclusion: hu may be administered in thalassemia major and intermediate patients to minimize or obviate the need for regular transfusion and concomitant iron overload. hu therapy appears to be safe and effective when administrated in thalassemia patients.