Hydroxyurea can eliminate transfusion requirements in children with severe beta thalassemia Hydroxyurea treatment in β-thalassemic children

Hydroxyurea (HU) enhances fetal hemoglobin (Hb) production. An increase in total Hb level has been repeatedly reported during HU treatment in patients with sickle cell disease and in several patients with β-thalassemia intermedia. Effects in patients with β-thalassemia major are controversial. We now report a marked elevation of total Hb levels with HU that permitted regular transfusions to be stopped in seven transfusion-dependent β-thalassemic children. The median follow-up was 19 + 3 months (range: 13-21). We conclude that HU can eliminate transfusional needs in children with β-thalassemia major, which could be particularly useful in countries like Algeria, where supplies of blood or chelating agents are limited. Corresponding author: mariane.demontal@nck.ap-hop-paris.fr Introduction Hydroxyurea (HU) promotes fetal hemoglobin (HbF) production via a reactivation of γ-genes, thanks to molecular mechanisms that are not yet elucidated. The clinical benefit induced by this compound in patients affected with sickle cell disease has been repeatedly demonstrated [1,2]. A significant benefit could also be expected in patients with βthalassemia, since the imbalance in globin chains could be ameliorated by the newly synthesized γ-chains being able to neutralize the excess α-chains, which could partially correct ineffective erythropoiesis. Clinical and hematological improvements have been reported in patients with thalassemia intermedia [3-8], but responses in patients with thalassemia major are controversial [5,9]. We have followed up seven transfusion-dependent β-thalassemic children, 6 of them with severe transfusional complications, and have treated them with hydroxyurea in the hope that this drug could reduce transfusional needs. Study design For personal use only. by guest on June 11, 2013. bloodjournal.hematologylibrary.org From