New therapies for sickle cell disease.

New and developing therapeutic agents for the treatment of sickle cell disease include hydroxyurea (an unlicensed experimental drug in most countries), omega-3 fatty acids, and the Gardos channel inhibitor ICA-17043. Anti-cellular adhesion therapy has considerable prospects; however, it has yet to be translated into clinical practice. For specific disease manifestations, pulmonary hypertension responds well to oral arginine, l-carnitine, and exchange blood transfusion therapy alone or in combination with other agents. Primary stroke prevention with transfusion therapy is now considered standard care. Oral iron chelators are administered increasingly instead of the more inconvenient parenteral desferrioxamine. Deferiprone is licensed in Europe and India, and deferasirox (ICL670) holds out important promise because it has not been shown to affect blood cell counts.