Hydroxyurea A Viable Option for Some Children with Sickle Cell Anemia

Sunday, December 06, 2015

CINCINNATI – A sickle cell disease study has found that the drug hydroxyurea is as effective as blood transfusions in children to reduce blood flow speeds in the brain, which is a key risk factor for stroke.

The findings appear in The Lancet on Dec. 6 and were presented at the American Society of Hematology meeting the same day. Cincinnati Children’s Hospital Medical Center helped lead the multi-institutional clinical trial and was the study’s medical coordinating center. 

Some children living with sickle cell disease are at increased risk of stroke. The risk is measured using a technique called transcranial doppler, which can determine speed of blood in the brain. Stroke risk is increased in children who have high transcranial doppler velocity measurements. 

Standard treatment for kids with sickle cell disease who are at high risk of stroke has been regular blood transfusions. The National Institutes of Health-supported study sought to answer whether hydroxyurea was no worse 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. 

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 Russell Ware, MD, PhD, one of the study’s principal investigators and director of the Division of Hematology at Cincinnati Children’s. “While blood transfusions are an effective option, there are questions about the long-term consequences of regular transfusions. Now families and healthcare 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.

Researchers from 26 sites recruited children ranging in age from 4 to 16 years old. People in TWiTCH received treatment through the study for a two-year period. The study authors indicated that the findings suggest that hydroxyurea could be effective at reducing risk of stroke, though this was not a primary goal of the study.

“No child should ever have to face the prospect of suffering through a stroke,” said Gary H. Gibbons, MD, director of the NHLBI. “Our institute is striving to achieve a stroke-free generation of children living with sickle cell disease. Studies like this are vital for moving us toward this worthwhile goal.”

About Cincinnati Children's

Cincinnati Children’s Hospital Medical Center ranks third in the nation among all Honor Roll hospitals in U.S. News & World Report’s 2015 Best Children’s Hospitals. It is also ranked in the top 10 for all 10 pediatric specialties, including a #1 ranking in pulmonology and #2 in cancer and in nephrology. Cincinnati Children’s, a non-profit organization, is one of the top three recipients of pediatric research grants from the National Institutes of Health, and a research and teaching affiliate of the University of Cincinnati College of Medicine. The medical center is internationally recognized for improving child health and transforming delivery of care through fully integrated, globally recognized research, education and innovation. Additional information can be found at www.cincinnatichildrens.org. Connect on the Cincinnati Children’s blog, via Facebook and on Twitter.