Fixed vs. Escalated Dosing of Hydroxyurea for Sickle Cell Anemia in Sub-Saharan Africa

Hydroxyurea (HU) modifies the natural history of sickle cell anemia in adults and children. Unfortunately, this agent is not routinely available for children with sickle cell anemia in sub-Saharan Africa, where rates of the illness are expected to increase significantly over the next 2 decades. Moreover, the optimal dosing and monitoring regimens are unknown for affected children in this region who do have access to HU. Prior studies have shown safety and efficacy of both fixed-dose and dose-escalation programs, but these strategies have not been previously compared with one another.

Now, investigators have conducted a randomized, double-blind, clinical trial (NOHARM MTD) involving 187 children with sickle cell anemia in Kampala, Uganda, who…