Our results are promising and justify a larger multicenter clinical trial to confirm that treating babies with hydroxyurea is safe and effective, said Jane S. Hankins, M.D., a physician at the St. Jude Comprehensive Sickle Cell Center and the studys lead author. If a larger trial supports our observations in the HUSOFT Extension, the treatment of sickle cell anemia will undergo a significant change.
This study is particularly encouraging because it suggests that we can treat babies with hydroxyurea for several years without side effects serious enough to limit the use of this drug, said Winfred Wang, M.D., St. Jude Comprehensive Sickle Cell Center director. Our aim is to make sickle cell anemia a survivable disease that doesnt significantly reduce a persons quality of life. Wang is the senior author of the paper in Blood.
A two-year pilot study of 21 babies with SCA, the original HUSOFT was designed to examine the feasibility of treating infants with liquid hydroxyurea; to determine the toxicity of this drug in babies; to assess hydroxyureas effects on fetal Hb levels; and to observe if this treatment could preserve spleen function. Patients received 20 milligrams/kilgrams of body weight/day (mg/kg/day) of hydroxyurea. All 21 patients who completed the initial study were enrolled by their parents into the HUSOFT Extension study. In that study, the dose of hydroxyurea was elevated from 20 to 30 mg/kg/day for an average of 4.0 years (range 2.1 6.0 years).
The aim of the HUSOFT Extension was to determine if this higher dose, given for an extended period of time, provided significant long-term benefits without causing unacceptable side effects in children ranging in age from 2.6 to 4.4 years (median age 3.4 years).
After four years of hydroxyurea therapy, the concentrations of Hb, HbF and the volu