For young sickle cell anemia (SCA) patients at the highest risk of stroke, a daily dose of the medication hydroxyurea provides management of the disease that is just as effective as monthly blood transfusions, without the issues of iron overload and antibody formation which are sources of morbidity in these children. That finding is the result of a recent clinical trial that was halted a year early by the National Heart, Lung, and Blood Institute (NHLBI) because its findings were so conclusive.

“Hydroxyurea clearly works as well as blood transfusions to lower the risk of a child with sickle cell anemia having a stroke. Only rarely is a study stopped early for success,” said Russell Ware, M.D. Ph.D., director of Hematology at Cincinnati Children’s Hospital Medical Center, which served as the study’s medical coordinating center. “This is a major advancement.”

The trial was conducted 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. It included children between the ages of 4 and 16 who were at the highest risk for stroke, and 121 children took part in the trial, with about half receiving blood transfusions and half receiving hydroxyurea.

The positive results researchers observed in the study open the door to more treatment options for clinicians trying to prevent strokes in children living with SCA, according to Ware.

Over the past decade, the laboratory and clinical effectiveness of hydroxyurea has been demonstrated in children and adults with SCA. Originally developed as a drug to treat cancer and infections, hydroxyurea boosts fetal hemoglobin production in patients with SCA, preventing the red blood cells from acquiring the sickle shape that fuels the many complications.

Hydroxyurea has been previously shown to be effective for a variety of sickle-related complications, but this was the first trial that demonstrated its benefits for children at increased risk of stroke.

Barry R. Davis, M.D., Ph.D., was principal investigator for the study’s data coordinating center at the University of Texas Health Science Center at Houston (UTHealth) School of Public Health. Serving as co-principal investigator for the study’s neurological core was Robert J. Adams, M.D., Medical University of South Carolina.

For more information, visit:

Cincinnati Children’s Hospital Medical Center – www.cincinnatichildrens.org;

University of Texas Health Science Center at Houston (UTHealth), www.uth.edu; and Medical University of South Carolina, http://academicdepartments.musc.edu/musc/.