Hydroxy Urea Therapy in Sickle Cell Anemia

Hydroxy Urea Therapy in Sickle Cell Anemia 

Several independent trials have shown that  

However, quite unexpectedly, the following observations were also made  

Although HU may mediate some of its clinical benefits through its positive effect on HbF expression, these data also suggest that it may, by a yet-to-be defined mechanism of action, modulate the clinical severity of SCA. One possible pathway is that among the two incriminated interacting cell partners (sickle erythrocyte and endothelial cell) involved in vaso-adhesion and occlusion, HU may also affect the phenotype status of endothelial cells so that its adhesogenic (structural) and/or vasoregulatory (functional) properties are modified in a favorable manner. 

Although the clinical benefits of HU therapy are unequivocal for a large number of patients, there are still some serious limitations  

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