A chemotherapy drug taken by mouth has been found to safely and effectively manage sickle cell disease in children, a new study shows.
Children taking hydroxyurea had fewer ER visits and spent fewer days in the hospital compared to kids not taking the drug, researchers reported April 17 in the journal Blood Advances.
“Our results reinforce that hydroxyurea, the most efficacious medicine available for sickle cell disease, continues to have really important benefits over time for pediatric patients,” lead researcher Dr. Paul George, a pediatric hematology/oncology fellow at Emory University School of Medicine in Atlanta, said in a news release.
What is sickle cell disease?
Sickle cell disease (SCD) is the most common inherited red blood cell disorder in the U.S., affecting an estimated 100,000 people, researchers said in background notes. One in every 365 Black children and one in every 16,300 Hispanic children are born with the disease.
The disorder causes abnormally shaped red blood cells that are sickle-shaped and sticky. If they become lodged in a person’s veins, they can block blood flow and cause organ damage, infection and episodes of severe pain.
What is hydroxyurea?
Hydroxyurea is taken once a day, and has been shown to reduce the frequency and severity of SCD pain crises, researchers said. The drug also decreases the need for blood transfusions, improves anemia, and reduces the risk of clots blocking blood vessels in the lungs.
The drug helps red blood cells stay round and flexible, rather than sickle-shaped, according to St. Jude Children’s Research Hospital.
Federal regulators currently recommend that hydroxyurea be offered to patients with severe sickle cell disease starting between 9 and 12 months of age, researchers noted.
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“Hydroxyurea has been a mainstay in sickle cell disease treatment for a long time, but was initially used as a chemotherapy, so there have always been some lingering fears about its safety and efficacy, especially for children,” researcher Dr. Wilbur Lam, a professor of pediatrics and biomedical engineering at Emory University, said in a news release.
“This study can provide some reassurance to patients and their families that this therapy, one of the most accessible for SCD, continues to be a safe option with a true benefit outside of a controlled setting,” Lam added.
How did the study work?
For the study, researchers tracked 2,147 children under age 18 with sickle cell disease treated at least three times between 2010 and 2021 at Children’s Hospital of Atlanta.
Of the children, 58 percent had used hydroxyurea. Average time on hydroxyurea was about five years, with 304 kids age 8 or older on continuous therapy with the drug.
Kids taking hydroxyurea consistently had fewer ER visits and days in the hospital, even after researchers accounted for the severity of their sickle cell disease and adherence to the drug therapy.
“Overall, hydroxyurea remained effective over time in these children,” George said. “However, one important takeaway from this study is that improvements in hemoglobin concentration – or reductions in anemia – were seen only in patients whose data indicated they were regularly taking the medication.”
Is hydroxyurea safe?
Yes, hydroxyurea has been extensively tested in clinical trials for sickle cell disease (SCD) and is a safe and well-established treatment.
Multiple studies, including the landmark Multicenter Study of Hydroxyurea (MSH) in adults published in 1995, showed a major reduction in the frequency of painful sickle cell crises in patients treated with hydroxyurea compared to placebo. Hydroxyurea has also been associated with decreased need for blood transfusions in sickle cell patients. Research has also confirmed the safety and efficacy of hydroxyurea in children as young as 9 months old, reducing pain crises, hospitalizations, and the risk of acute chest syndrome (ACS).
Long-term studies have also suggested that continually using hydroxyurea use can be safe and may even reduce death risk in individuals with sickle cell disease.
