Cookies on this website
We use cookies to ensure that we give you the best experience on our website. If you click 'Continue' we'll assume that you are happy to receive all cookies and you won't see this message again. Click 'Find out more' for information on how to change your cookie settings.

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

DOI

10.1196/annals.1345.031

Type

Journal article

Journal

Ann N Y Acad Sci

Publication Date

2005

Volume

1054

Pages

250 - 256

Keywords

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