Potential alternatives to erythrocyte transfusion in hemoglobinopathies: hydroxyurea (HU), erythropoietin (EPO), butyrate derivatives, blood substitutes

Transfusion is associated with improvement of life expectancy in beta thalassemia and sickle cell disease (SCD). Bone marrow transplantation concerns only a few patients. Among potentially useful therapeutic agents which can induce fetal hemoglobin (HbF) production, hydroxyurea (HU) stands out in particular for SCD. In our sickle cell center, 10 patients with SCD received HU for chronic leg ulcer with good results in terms of healing (7 among 10, who required no additional transfusion). A few patients, most of them over 30 years, received HU for chronic organ failure at the onset. HU on long term therapy over one year improved general status, Hb level, in 7 patients with beta thalassemia intermedia. A major challenge in the coming years will be the effective evaluation of clinical efficiency and the comparison of risks and benefits of such compounds versus transfusion.