Most sickle cell patients who have increased levels of HbF experience milder forms of the disease than do patients with normal or low levels of HbF. Therefore, the focus of molecular treatments for sickle cell is to increase the level of fetal hemoglobin (HbF).
The only drug currently approved by the FDA, which is used to induce increased production of HbF, is hydroxyurea. Hydroxyurea is a myelosuppresent and a ribonucleotide reductase inhibitor. However, the mechanism of hydroxyurea's influence in HbF production is not well understood.
Hydroxyurea may also contribute to reduction of vaso-occlusion.
