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Prepublished online as a Blood First Edition Paper on April 17, 2003; DOI 10.1182/blood-2003-01-0117.

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Blood, 15 August 2003, Vol. 102, No. 4, pp. 1529-1530

RED CELLS
Brief report

Hydroxyurea can eliminate transfusion requirements in children with severe {beta}-thalassemia

Mohamed Bradai, Mohand Tayeb Abad, Serge Pissard, Fatima Lamraoui, Laurent Skopinski, and Mariane de Montalembert

From the Service d'Hématologie, Hôpital Franz Fanon, Blida, Algeria; Laboratoire de Génétique Moléculaire and Institut National de la Santé etdela Recherche Médicale (INSERM) U 468, Hôpital Henri Mondor, Créteil, France; and Service de Pédiatrie Générale, Hôpital Necker-Enfants Malades, Paris, France.

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 {beta}-thalassemia intermedia. Effects in patients with {beta}-thalassemia major are controversial. We now report a marked elevation of total Hb levels with HU that permitted regular transfusions to be stopped in 7 children with transfusion-dependent {beta}-thalassemia. The median follow-up was 19 ± 3 months (range, 13-21 months). We conclude that HU can eliminate transfusional needs in children with {beta}-thalassemia major, which could be particularly useful in countries such as Algeria, where supplies of blood or chelating agents are limited.


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