Many have requested information on currently approved medications for sickle cell. Hydroxyurea is still the gold standard for treatment and is the only medication we can say will make you live longer with sickle cell. BABY-HUG studied the effectiveness of hydroxyurea on babies, specifically looking at reducing pain episodes and organ damage. It does cause various side effects (which is why we need to monitor your labs to make sure it’s having the effect we want) and can’t be used for all patients, so it is exciting to have newers options available!
Endari was the first medication approved for sickle cell in almost 20 years! Oxbryta and Adakveo were both approved at the end of 2019 and address different aspects of sickle cell. Many think it is only the sickled cells that cause the occlusion in the vessel, leading to pain, however that is only the first problem. After the cells sickle, we have white blood cells, platelets and other inflammatory cells attaching to the blocked vessel, which adds more insult to injury, making the entire problem worse. Adakveo helps address the inflammatory component of sickle cell while Oxbrya helps prevent hemolysis (red cell breakdown) in the first place.
Let me know what questions you may have!
theHemeNP 