NHLBI IN THE PRESS
Sickle cell disease drug proves safe, effective in African children

A daily hydroxyurea pill has proven safe and effective for young children living with sickle cell disease in sub-Saharan Africa, where the condition is far more prevalent, according to the reported results of REACH, a large multinational, NHLBI-funded clinical trial.

The researchers tested daily hydroxyurea treatment in 606 children between the ages of 1 and 10 years old. The children took a pill each day for six months, followed by increases in the daily dosages based on their health status and weight. Initial doses varied between 15-20 mg/kg a day.

The study results, published in The New England Journal of Medicine, were presented at the American Society of Hematology's annual meeting in San Diego.

The clinical trial determined that hydroxyurea therapy is feasible to use and safe for children in sub-Saharan Africa. Compared to pre-treatment levels, hydroxyurea use was linked to reduced rates of sickle cell pain by an average of 55 percent, infections by 38 percent, malaria by 51 percent, transfusions by 67 percent, and death by 70 percent.

“Hydroxyurea was safe and offered many benefits to these young patients, including improved anemia, fewer sickle cell pain events, less malaria, and better survival,” Russell Ware, MD, PhD, the study's senior investigator and a physician-scientist at the Cincinnati Children's Cancer and Blood Diseases Institute, said in a press release.