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Children's National Researchers Contribute to National Sickle Cell Study

The Drug Hydroxyurea Prevents Pain Crises and is Shown Safe in Infants and Small Children

Washington, DC – Lori Luchtman-Jones, MD, Division Chief of Hematology at Children’s National Health System, Caterina Minniti, MD, with the Cardiovascular and Pulmonary Branch of the National Heart, Lung and Blood Institute (NHLBI), National Institutes of Health (NIH), and Brenda Martin, PNP, nurse practitioner at Children’s National, were among the collaborators in a multi-institution study that demonstrated the safety and efficacy of the drug hydroxyurea in infants aged 9 – 17 months with sickle cell disease. The drug was shown to reduce the number of painful sickle cell-related crises, an improvement in care for patients with the disease.

“This drug could be a game-changer for children with sickle cell,” said Dr. Luchtman-Jones. “This study showed that hydroxyurea was safe and effective and can be considered for very young children with the disease, even before they have their first painful crisis, so we can prevent or at least reduce the frequency and severity of the crises associated with sickle cell disease.”

Hydroxyurea is often used in adults with the disease, but until this study of such young patients – the largest of its kind – its effectiveness had not been tested adequately in infants. Hydroxyurea in adults is known to increase fetal hemoglobin, which reduces or prevents sickle red blood cell hemoglobin from becoming rigid and blocking blood flow to tissues. However, there has been concern about whether hydroxyurea might cause serious long-term health problems and about the drug’s effectiveness in infants and children if given before they had evidence of typical sickle cell crises.

“For years we have focused on medications that control pain in babies and children with sickle cell," said Dr. Luchtman-Jones. "So testing a drug that prevents pain crises by addressing the disease mechanism and finding it safe and effective for infants, is an extremely important advance in caring for our smallest patients.”

Sickle cell anemia is an inherited disorder in which the hallmark, sickle-shaped, red blood cells block blood flow to tissues, causing severe pain crises and organ damage. Children’s National provides care to more than 1,300 children and teens with sickle cell disease living in the Washington, DC, metro area.

Published recently in The Lancet, the Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) study, was a phase III, randomized, double-blinded, placebo-controlled study of 193 infants conducted at 14 U.S. clinical centers, including Children’s National, and funded by the NHLBI.

Researchers found that hydroxyurea reduced the number of painful vaso-occlusive complications in those who were given the drug. As a group, the infants and toddlers in the study who received hydroxyurea (revealed once data collection was complete), experienced half the number of all painful events, compared to those who did not receive the drug. Additionally, the group receiving the drug experienced certain painful events (including inflammation of hands or feet, or acute chest syndrome) to a significantly lesser degree − 80 percent less.

The families of 163 patients of the original 193 group in the BABY HUG group have agreed to continue in a follow-up, longitudinal study, to investigate the longer term impact of the drug on children.

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