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Single and combination drug therapy for fetal hemoglobin augmentation in hemoglobin E-beta 0-thalassemia:

Sylvia T Singer1, Frans A Kuypers, Nancy F Olivieri

  • 1Children's Hospital & Research Center at Oakland, 747 52nd St., Oakland, CA 94609, USA. tsinger@mail.cho.org

Annals of the New York Academy of Sciences
|December 13, 2005
PubMed

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.

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