Clinical and Hematological Response to Hydroxyurea in a Patient with Hb Leporbp-Thalassemia

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Abstract
The possibility of increasing Hb F in vivo using drugs like 5-azacy-tidine, hydroxyurea, and butyrate has been established. However, in many cases this does not entail an increase in total hemoglobin. We report on a patient with Hb Lepore/β-thalassemia being treated with hydroxyurea (30 mg/Kg/day) because of the presence of erythroid extra-medullary masses with severe neurological abnormalities. During therapy the patient showed a remarkable improvement in neurological signs due to the reduction in extra-medullary masses, a significant increase in both total hemoglobin (from 5.8 to 9.7 g/dl) and Hb F (from 4.9 g/dl to 9.1 g/dl). The marked improvement in hemoglobin level in our patient with Hb Lepore/β-thalassemia suggests γ-globin gene activation due to the DNA structure determined by the crossover event.