Hydroxyurea treatment can greatly benefit children with sickle cell anemia, study reveals

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Clinical studies led by Indiana University School of Medicine investigators and their collaborators in Uganda have revealed that hydroxyurea significantly reduces infections in children with sickle cell anemia. Their latest findings add to strong evidence of hydroxyurea’s effectiveness and may ultimately reduce child mortality in Africa, the continent most burdened by the disease.

The group’s research was recently published in the journal the bloodrevealed that hydroxyurea treatment significantly reduced serious or invasive infections, including malaria, bacteremia, respiratory infections, and gastroenteritis, by 60% in children with sickle cell anemia in Uganda.

Our investigation provides a strong rationale for the use of hydroxyurea in children with sickle cell anemia in Africa. Given the high rate of infection in this region, we hope that our evidence will support ministries of health and encourage them to continue expanding access to hydroxyurea for young patients who could greatly benefit from treatment.”

Dr. Chandy John, the Ryan White Professor of Pediatrics at the IU School of Medicine and co-principal investigator of the latest study.

Sickle cell anemia is a genetic blood disorder that changes the structure of red blood cells and affects oxygen delivery throughout the body, increasing susceptibility to serious health complications and life-threatening infections. According to the World Health Organization, more than 300,000 children worldwide are born with sickle cell disease each year, with a high prevalence in African countries.

While hydroxyurea has been approved by the US Food and Drug Administration as a treatment for sickle cell disease in children since 2017, its accessibility and acceptance in Africa is relatively limited. As hydroxyurea has become more recognized for its effectiveness in treating sickle-cell-related complications in African countries, John and his colleagues have noticed a gap in knowledge about the treatment’s effect on infection. This led the research group to incorporate hydroxyurea treatment and analysis into zinc for infection prevention in sickle cell anemia, their established clinical trial.

During the study, researchers examined the effects of hydroxyurea on 117 children in Uganda and focused on different infections. After hydroxyurea treatment, results showed a significant reduction in the incidence of these infections. Additionally, eight of the nine deaths in the trial were children whose parents refused hydroxyurea treatment. The only infant death on hydroxyurea treatment occurred four days after starting treatment, giving insufficient time for hydroxyurea to take effect.

Of the five children whose cause of death was known, five died of infectious causes. The high mortality rate in the study, despite expert clinical care by study staff, provides further evidence of the urgent need for additional interventions to reduce mortality in children with sickle cell disease in Africa.

“Infection usually precedes other complications associated with sickle cell anemia and often results in hospitalization and death,” said Dr. Ruth Namazi, the site’s principal investigator, is the study’s first author and a lecturer in the Department of Pediatrics and Child Health at Makerere University in Uganda. “We believe that including hydroxyurea treatment as a standard of care for sickle cell anemia across Africa will not only reduce infections but more importantly save countless lives.”

Additional IU researchers who contributed to the study include Caitlin Bond, Andrea Conroy, Divyadyuti Dutta and Michael Goings. They cooperated with the Namazis; Dr. Robert Opoka of Global Health Uganda; Dr. Abner Tagula of Jinja Regional Referral Hospital; Jeong Hun Jang of Yonsei University; and Dr. Russell E. Ware of Cincinnati Children’s Hospital Medical Center.


Journal Reference:

Namazi, R., etc. (2024). Hydroxyurea reduces infections in children with sickle cell anemia in Uganda. the blood. doi.org/10.1182/blood.2023021575.

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