A sickle cell disease study has found that the drug hydroxyurea is as effective as blood transfusions at reducing blood flow speeds in children’s brains, which is a key risk factor for stroke.
The findings, which are part of a multi-institutional study that included researchers from The University of Texas Health Science Center at Houston (UTHealth) School of Public Health Coordinating Center for Clinical Trials, appeared in The Lancet on December 6 and were presented at the American Society of Hematology meeting the same day.
Some children living with sickle cell disease are at increased risk of stroke. The risk is measured using a technique called transcranial doppler (TCD), which can determine the speed of blood flow in the brain. Stroke risk is increased in children who have high TCD velocity measurements.
Regular blood transfusions are standard treatment for kids with sickle cell disease who are at high risk of stroke. The National Institutes of Health-supported study sought to answer whether hydroxyurea was as good as or better than blood transfusions. The Transcranial Doppler with Transfusions Changing to Hydroxyurea (TWiTCH) study was stopped early due to positive preliminary results in November 2014.
Hydroxyurea is the only drug approved by the Food and Drug Administration to treat sickle cell disease.
“Our Coordinating Center is proud to have contributed to this important trial that likely will change the clinical care of children with sickle cell anemia and elevated TCD velocities,” said Dr. Barry R. Davis, principal investigator for the Coordinating Center for Clinical Trials at UTHealth School of Public Health.
Researchers supported by the NIH’s National Heart, Lung, and Blood Institute (NHLBI) studied 121 children and divided them into two groups: one that received transfusions and one that was transitioned from transfusions to daily doses of hydroxyurea.
“We wanted to provide the medical community with another way to help treat children who are at the greatest risk of stroke,” said Dr. Russell Ware, one of the study’s principal investigators and director of hematology at Cincinnati Children’s Hospital Medical Center. “While blood transfusions are an effective option, there are questions about the long-term consequences of regular transfusions. Now families and health care providers have an additional treatment option with hydroxyurea.”
Part of the challenge is that children who receive regular blood transfusions must also be managed to avoid the danger of iron overload. Chelation, or iron-reduction, therapy is needed for those receiving transfusions, according to Ware.
-Courtesy of Cincinnati Children’s Hospital Medical Center