Beginning as early as age 2, children with sickle cell anemia (SCA) can suffer from significant end-organ dysfunction related to their disease. Hydroxyurea is an antimetabolite agent that is known to increase levels of fetal hemoglobin (HbF) in pediatric and adult patients. The HUSOFT study was the first to show the feasibility and safety of hydroxyurea in infants with SCA after 2 years of treatment.¹ At the completion of that trial, however, little remained known about the long-term effects of higher doses of hydroxyurea or its possible efficacy in reducing irreversible end-organ dysfunction related to SCA, leading to this extension study.

To assess the safety and efficacy of long-term hydroxyurea with dose escalation in young children with SCA.

Prospective, multicenter, open-label, single-arm study at 5 US centers from 1996 to 1997.