The role of hydroxyurea in the management of sickle cell disease

Sickle cell disease (SCD) is one of the most common genetic diseases with some 250,000 new births each year. Most patients suffer intermittent pain crises and life-threatening events while life expectancy is considerably reduced. Until the last decade management was purely preventative or supportive aimed at symptom control. Apart from stem cell transplant, there is no cure but the oral chemotherapeutic drug hydroxyurea (HU) has now established a role in ameliorating the disease and improving life expectancy for most patients. There are side effects and risks of HU treatment in SCD but for moderate and severely affected patients, the benefits can be significant.