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

Abstract

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.

• Authors: Mohamed Bradai, Mohand Tayeb Abad, Serge Pissard, Fatima Lamraoui, Laurent Skopinski, Mariane de Montalembert
• Journal: Blood (Blood) Vol. 102 Issue 4 Pg. 1529-30 (Aug 15 2003) ISSN: 0006-4971 [Print] United States
• PMID: 12702505 (Publication Type: Clinical Trial, Journal Article)

Chemical References

• Hemoglobins
• Hydroxyurea

Topics

• Adolescent
• Blood Transfusion
• Child
• Child, Preschool
• Female
• Follow-Up Studies
• Hemoglobins (analysis, biosynthesis, drug effects)
• Humans
• Hydroxyurea (therapeutic use)
• Male
• Treatment Outcome
• beta-Thalassemia (blood, drug therapy, therapy)