Response of patients with intermediate beta-thalassemia to treatment with hydroxyurea

Abstract

Background: Hydroxyurea is a chemotherapeutic agent for treatment of cancer. This drug induces globin-γ synthesis, so it could be used for treatment of thalassemia. Several studies have been shown that treatment with hydroxyurea increases Hb and HbF levels in patients with intermediate betathalassemia. However, the efficiency of hydroxyurea treatment in patients with beta-thalassemia is unclear. In the present study, clinical response of these patients to the drug was investigated. Methods: In this prospective study, the samples were patients with beta-thalassemia intermedia admitted to Sayed-al-Shohada hospital, Isfahan, Iran, during the years 2011-13. Efficiency of hydroxyurea in 46 patients was studied by determining the changes of Hb and HbF levels before and after one year of treatment with the drug. Treatment was performed using 500 mg capsule with dosage of 20 mg/day/kg. Patients were monitored for side effects, too. Findings: After treatment, the means of Hb and HbF levels increased at a rate of 0.47 ± 1.12 g/dl and 6.04 ± 1.43 percent, respectively; where the first was statistically significant, but the latter was not. Use of drug improved the quality of the patient's condition and there was no side effect. Conclusion: According to our results, it is suggested that treatment with hydroxyurea could be effective in majority of patients with intermediate beta-thalassemia.