St. Jude Children’s Research Hospital investigators have shown that using the drug hydroxyurea to boost average fetal hemoglobin levels above 20 percent in children and teenagers with sickle cell anemia was associated with at least a two-fold reduction in hospitalization for any reason.
The findings should help settle the debate about how to optimize hydroxyurea for treatment of sickle cell disease in young people. Rather than calculating a standard dose of hydroxyurea based on patients’ weight, researchers used a dose-escalation approach to determine the maximum tolerated dose for each of the 230 St. Jude patients enrolled in the study.
“Our analysis showed that using this approach, hospitalizations for the average patient fell to less than one every couple of years rather than four to six annually,” said lead author Jeremie Estepp, M.D.
, an assistant member of the St. Jude Department of Hematology
. “This frees children from the fevers, pain and other symptoms of this disease and gives them and their families more chances to enjoy childhood and adolescence.”