Adult Sickle Cell Anemia Drug Found Safe and Effective in Infants

Hydroxycarbamide (formerly hydroxyurea) is a medication taken once a day and approved by the Food and Drug Association for use in adults with sickle cell anemia. In adults, it reduces pain and pneumonia and improves life expectancy. 

The study, called BABY HUG, enrolled nearly 200 infants age 9-18 months from SUNY Downstate and other sickle cell care centers to determine whether giving hydroxycarbamide early in life is safe and effective. The results of the study, published in the most recent issue of the medical journal The Lancet, showed that babies who received hydroxycarbamide experienced less pain, had fewer hospitalizations and bouts of pneumonia, and needed about half as many transfusions compared to those who did not receive the treatment.

Scott Miller, MD, a pediatric hematologist who is professor of clinical pediatrics at Downstate and a member of the BABY HUG study team, has been recommending hydroxycarbamide therapy to pediatric sickle cell patients for years. He says, “Based on several small studies showing use in children to be safe and similarly beneficial, we have over the last 10 years prescribed hydroxycarbamide to many children who have complications due to sickle cell disease, with much success.”

He added, “The BABY HUG results should encourage all doctors who see children with sickle cell anemia to become familiar with how to prescribe hydroxycarbamide, and treatment should be strongly considered for children who have any problems. In fact, I wonder if everyone with sickle cell anemia shouldn’t be taking hydroxycarbamide.”

Dr. Miller notes that a common side effect is lowering of the white blood count and recommends that children taking hydroxycarbamide should have their blood checked once a month to avoid difficulties.  

Also participating in the study were St. Jude Children’s Research Hospital in Memphis, Children’s National Medical Center in Washington, Johns Hopkins University School of Medicine in Baltimore, Howard University College of Medicine, University of Mississippi Medical Center, Duke University Medical Center, University of Texas Southwestern Medical Center at Dallas, Medical University of South Carolina, University of Miami, Emory University School of Medicine, Children’s Hospital of Michigan, University of Alabama at Birmingham, Medical College of Georgia, the National Heart, Lung and Blood Institute, and the Clinical Trials & Surveys Corporation.


SUNY Downstate Medical Center, founded in 1860, was the first medical school in the United States to bring teaching out of the lecture hall and to the patient’s bedside. A center of innovation and excellence in research and clinical service delivery, SUNY Downstate Medical Center comprises a College of Medicine, Colleges of Nursing and Health Related Professions, a School of Graduate Studies, a School of Public Health, University Hospital of Brooklyn, and an Advanced Biotechnology Park and Biotechnology Incubator.

SUNY Downstate ranks ninth nationally in the number of alumni who are on the faculty of American medical schools.  More physicians practicing in New York City have graduated from SUNY Downstate than from any other medical school.