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Patients with hemoglobin E (Hb E)-beta 0-thalassemia, one of the most common hemoglobinopathies worldwide, could benefit from drugs that increase fetal and total hemoglobin levels and thereby decrease the need for transfusions. The long-term clinical outcome of such therapy, its hematologic effects, and which patients are likely to benefit from treatment are unknown. Consequently, the use of such drugs for Hb E-beta 0-thalassemia is limited, and countries where resources for safe and regular transfusion are scarce cannot benefit from them. In a multicenter trial of 42 patients treated with hydroxyurea for two years, almost half the patients demonstrated a significant increase in steady-state hemoglobin level. Drug toxicity was minimal. Combined treatment of hydroxyurea with erythropoietin benefited selected patients, but the addition of sodium phenyl butyrate was ineffective. After 5 years of follow-up, a subset of patients remained off transfusions. Hydroxyurea should be considered for a subset of Hb E-beta 0-thalassemia patients.

Original publication




Journal article


Ann N Y Acad Sci

Publication Date





250 - 256


Blood Transfusion, Combined Modality Therapy, Drug Therapy, Combination, Erythropoiesis, Erythropoietin, Facial Bones, Fetal Hemoglobin, Gene Expression, Genotype, Globins, Hematopoiesis, Extramedullary, Hemoglobin E, Humans, Hydroxyurea, Phenylbutyrates, Radiography, Recombinant Proteins, Splenectomy, Splenomegaly, Treatment Outcome, beta-Thalassemia