Fewer serious complications. Fewer hospitalizations and blood transfusions. Better growth and development. And a markedly lower risk of death from the complications of sickle cell anemia.
These are the benefits documented from 10 years of continuous hydroxyurea treatment provided in the NOHARM trial to a group of young children in Uganda, which has one of the world’s largest number of people living with the painful disorder known for causing sickle-shaped red blood cells. These improved outcomes were highlighted May 27, 2026, in a report published by The New England Journal of Medicine.
Russell Ware, MD, PhD, director of the Division of Hematology and the Global Health Center at Cincinnati Children’s, was the lead author of the report. He has been working for years with researchers and clinicians across sub-Saharan Africa to demonstrate the safety and effectiveness of low-cost hydroxyurea treatments.
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“In a low-resource environment like sub-Saharan Africa where about 50% of children with sickle cell anemia will die by age 10 years, we have reduced the death rate to about 1-2% per year. That’s at least an 80% improvement over the natural history of this condition in this region,” Ware says.
In sub-Saharan Africa, an estimated 300,000 to 400,000 babies are born with sickle cell disease. Far fewer infants are born with sickle cell disease in the United States, but their life expectancy now exceeds 50 years of age. That’s because the U.S. offers widespread mandatory newborn screenings, and children have access to multiple forms of treatment, including penicillin provided early in life, hydroxyurea, regular blood transfusions and other forms of care to manage organ damage. In recent years, new and expensive gene therapies have offered cures to some people with the disease.
Hydroxyurea – an older medication initially used to treat cancer and HIV is available in Africa for anywhere from 10 cents to $1 per capsule. The drug prompts the body to produce fetal hemoglobin (HbF), which helps prevent red blood cells from sickling. Regular use of the drug reduces the frequency of painful crises, episodes of acute chest syndrome, and the need for blood transfusions.
In the new NEJM report, Ware and colleagues add that providing the “maximum tolerated dose” is notably more effective than smaller doses and still pose no major long-term safety concerns. The researchers found that many patients can tolerate doses as high as 30 mg/kg/day versus initial “fixed” doses of 15-20 mg/kg/day used earlier in their studies.
“These findings matter globally because they show that a proven sickle cell therapy can work safely and effectively even in settings complicated by malaria, malnutrition, and limited access to care,” Ware says.
