[Hydroxyurea treatment in patients affected with sickle cell anemia: efficacy and safety]. / Traitement des patients drépanocytaires par hydroxyurée: efficacité et tolérance.

ABSTRACT

Hydroxyurea is the unique drug having demonstrated an efficacy in preventing recurrences of painful crises, acute chest syndromes and in reducing transfusional needs in patients severely affected with sickle cell disease. However, there is a wide variation in the clinical response to hydroxyurea in sickle cell patients, with children generally experiencing greater benefits than adults. Short- and middle-term tolerances are good. Our uncertainties about long-term tolerance are mainly that we do not know the consequences of the drug on ulterior fertility in boys treated early and for long periods. Hydroxyurea has just been licensed for sickle cell adults and children in Europe. Its prescription for the moment must be restricted to severely affected patients, enrolled in long-term follow-up protocols.