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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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Submitted January 14, 2003
Accepted April 1, 2003

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*

Service d'Hematologie, Hopital Franz Fanon, Blida, Algeria
Laboratoire de Genetique Moleculaire and U 468, Hopital Henri Mondor, Creteil, France
Service de Pediatrie Generale, Hopital Necker-Enfants Malades, Paris, France

* Corresponding author; email: mariane.demontal{at}nck.ap-hop-paris.fr.

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 seven transfusion-dependent {beta}-thalassemic children. The median follow-up was 19 ± 3 months (range: 13-21). We conclude that HU can eliminate transfusional needs in children with {beta}-thalassemia major, which be could particularly useful in countries like Algeria, where supplies of blood or chelating agents are limited.


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