POTENTIAL ALTERNATIVE TREATMENTS TO BLOOD -TRANSFUSION IN HEMOGLOBINOPATHIES - HYDROXYUREA (HU), ERYTHROPOIETIN (EPO), BUTYRATE-DERIVED PRODUCTS, 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 ag ents which can induce fetal hemoglobin (HbF) production, hydroxyurea ( HU) stands out in particular for SCD. In our sickle cell center, 10 pa tients 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 impr oved general status, Hb level, in 7 patients with beta thalassemia int ermedia.A major challenge in the coming years will be the effective ev aluation of clinical efficiency and the comparison of risks and benefi ts of such compounds versus transfusion.