"Major advancement" for sickle cell patients

Jan 27, 2015

In what's being called a "major advancement," Cincinnati Children's Hospital Medical Center and other researchers around the country say young sickle cell anemia patients have a safer way to manage their disease.

Russell Ware, MD PhD,director of Hematology at Cincinnati Children's says the standard of care is lifetime blood transfusions for those at risk of stroke. But a clinical trial shows the medicine hydroxyurea  is just as good as blood transfusions without the cost and side effects.

Sickle Cell Anemia is an inherited blood disorder where blood cells change from a healthy round shape to a stretched out sickle shape.

Hydroxyurea works because it changes the way children make blood. Dr. Ware says he blood cells are rounded and stay rounded.

The trial was done at 25 medical centers in the U.S. and Canada, comparing the standard therapy of monthly blood transfusions with the alternative of taking a daily pill of hydroxyurea.  The National Heart, Lung, and Blood Institute (NHLBI) halted its recent clinical trial a year early because its findings were so conclusive.