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Effectiveness and toxicity of hydroxyurea in children with sickle cell anemia

Sickle cell anemia is a genetic disease characterized by a high morbimortality rate, it is considered as the most serious among all sickle cell diseases. The most effective therapeutic options available nowadays for the treatment of this hemoglobinopathy are bone morrow transplantation (BMT) and hydroxyurea (HU). BMT is considered a high risk procedure due to the different complications and significant mortality rates. The use of HU for children with sickle cell anemia has reduced the clinical complications and given a significant increase in life expectancy by augmenting the fetal hemoglobin levels and hemoglobin concentrations and reducing cytomegalovirus, as well as reducing hemolysis and vaso-occlusive events. Thus, HU is considered the best therapeutic option currently available. However, as HU has been identified as a potentially carcinogenic drug, there are questions related to the benefits and toxicities when it is used over long periods of time. This work aimed at evaluating, through a review of the literature, the risks, benefits and adverse effects of the use of hydroxyurea in children.

Sickle cell anemia; hydroxyurea; pediatric population


Associação Brasileira de Hematologia e Hemoterapia e Terapia Celular R. Dr. Diogo de Faria, 775 cj 114, 04037-002 São Paulo/SP/Brasil, Tel. (55 11) 2369-7767/2338-6764 - São Paulo - SP - Brazil
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