A 10-year study in sub-Saharan Africa revealed that a higher dose of the oral medication Hydroxyurea improved survival and growth outcomes in children with sickle cell anemia.
RT’s Three Key Takeaways:
- Mortality Reduction: Children with sickle cell anemia in sub-Saharan Africa saw an 80% decrease in mortality when treated with hydroxyurea compared to those not on the treatment.
- Clinical Outcomes: A maximum tolerated dose of the medication led to fewer hospitalizations, blood transfusions, and severe pain episodes.
- Growth and Development: Long-term use of hydroxyurea resulted in significant improvements in height and weight, suggesting better nutrition outcomes for pediatric patients.
A decade-long study found that hydroxyurea significantly improved survival rates for children with sickle cell anemia in sub-Saharan Africa, according to data published in the New England Journal of Medicine.
The findings, involving researchers from the Indiana University (IU) School of Medicine and global healthcare collaborators, suggest the treatment should be considered standard care for children with the inherited blood disorder.
Sickle cell anemia occurs when abnormally shaped red blood cells block oxygen from traveling through the body, which can lead to severe pain, delayed growth, infections, and other life-threatening complications. While hydroxyurea is a common treatment in high-resource regions like the US, it has been underutilized in sub-Saharan Africa, where the disease is most prevalent.
The research team examined the effects of the oral medication from 2014 to 2024. Through the Novel Use of Hydroxyurea in an African Region with Malaria (NOHARM) trial and subsequent studies, investigators compared a lower fixed dose against a maximum tolerated dose. The findings showed the higher dose resulted in a reduction of severe pain episodes, hospitalizations, and the need for blood transfusions.
“On the maximum tolerated dose of hydroxyurea, African children with sickle cell anemia survive and thrive,” said Chandy John, professor of pediatrics at the IU School of Medicine.
The study confirmed a significant reduction in mortality. The mortality rate for children in the NOHARM study was 8.7%, compared to a 43.3% mortality rate for children under 10 with sickle cell anemia in a separate study across five African countries.
“This reveals an 80% decrease in mortality for children on hydroxyurea as compared to children with sickle cell anemia in Africa not on, or not uniformly on, the treatment,” said John, co-principal investigator of the NOHARM studies.
Researchers also found that years of treatment led to substantial improvements in both height and weight, signaling improved nutrition outcomes. The project was a collaboration between the IU School of Medicine, Cincinnati Children’s Hospital Medical Center, Makerere University, and other international partners.
“The project has been a true collaboration from the beginning,” said John, director of the Ryan White Center for Pediatric Infectious Diseases and Global Health.
