Sickle Cell Disease - Highlights

Highlights

Drug Approval

Deferasirox (Exjade) has been approved for treatment of iron overload caused by frequent blood transfusions. The drug is approved for patients over age 2. Deferasirox will make it much easier for patients to tolerate iron chelation therapy. Chelation therapy currently requires an infusion pump and takes many hours. With deferasirox, patients mix the pills in liquid and drink the medicine once a day.

Drug Research

Hydroxyurea (Hydrea, Droxia), a drug used to prevent painful sickle cell attacks in adults and adolescents, may also help young children. A 2005 study reported that infants who received liquid hydroxyurea by mouth for several years showed improvements in height, weight, and spleen function. The drug also reduced episodes of acute chest syndrome. Most children in the study took the drug for at least 4 years.

Blood Transfusions

Regular blood transfusions may help children with sickle cell anemia regain weight and height, suggests a recent study. Patients in the Stroke Prevention for Sickle Cell Anemia trial were followed for 2 years. Last year, researchers reported that regular blood transfusions reduce the risk of stroke in high-risk children.

Arginine and Pulmonary Hypertension

The lack of the amino acid arginine may cause pulmonary hypertension, a serious lung disorder that is a complication of sickle cell disease. Pulmonary hypertension is high blood pressure in the arteries of the lungs and can be fatal. An important study in the Journal of the American Medical Association reported that patients with sickle cell disease who had low arginine levels were 3.6 times more likely to die than patients with high arginine levels. Doctors are working on developing new blood tests and drugs to better detect and treat reduced arginine levels. At this time, experts do not recommend that patients take arginine nutritional supplements.