A study in The New England Journal of Medicine found that 86% of children with sickle cell anemia who were given an escalated dose of hydroxyurea had reached the primary outcome thresholds of hemoglobin and fetal hemoglobin concentrations at trial closure, compared with 37% of those in the standard fixed-dose group. The findings, based on data involving 187 children in Uganda, also showed that those in the dose-escalation group needed fewer key medical interventions and had fewer sickle cell-related adverse events, compared with the fixed-dose group.
Dose escalation in pediatric sickle cell anemia examined
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