Successful Use of Hydroxyurea in β-Thalassemia Major

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Abstract
Hydroxyurea has been used in the treatment of sickle cell anemia to elevate hemoglobin levels and reduce clinical complications.1 The potential usefulness of hydroxyurea in the thalassemia syndromes is considerably less clear.1,2 In patients with β-thalassemia intermedia, the hematologic response to hydroxyurea alone or in combination with recombinant human erythropoietin is controversial,3-5 and little is known about the effectiveness of long-term therapy. The standard therapeutic approach to β-thalassemia major still relies on regular blood transfusions and the use of iron chelators. In this report, we describe the clinical and hematologic response of a 20-year-old man with transfusion-dependent β0-thalassemia (homozygous for a nonsense mutation at codon 39) to treatment with hydroxyurea.