Sickle cell anemia is a genetic red blood cell disorder in which the patient lacks enough healthy red blood cells to carry oxygen throughout the body. Hydroxyurea is an antineoplastic medication used to treat sickle-cell disease. This study aims to evaluate the effectiveness of hydroxyurea for treating sickle cell anemia in children with coexisting conditions like malnutrition and malaria.

This study included a total of 606 children aged 1-10 years with sickle cell anemia in four sub-Saharan nations. The participating children were assigned to receive hydroxyurea at a dose of 15 to 20 mg per kg body weight. The primary outcomes of the study were feasibility, safety, and benefits of hydroxyurea.

At 3 years of treatment, the retention rate among 606 children who completed screening and began the treatment was 94.2%. Hydroxyurea therapy resulted in increased levels of both hemoglobin and fetal hemoglobin. Treatment-related toxic events occurred in 5.1% of the participants. The use of hydroxyurea was also associated with a decreased rate of clinical adverse events, including vaso-occlusive pain, nonmalaria infection, malaria, transfusion, and death.

The research concluded that hydroxyurea treatment was safe and feasible in children with sickle cell anemia, and it reduced the incidence of vaso-occlusive events, malaria, transfusions, infections, and death.

Ref: https://www.nejm.org/doi/full/10.1056/NEJMoa1813598