Source
The New England Journal of Medicine
Sickle cell anemia is a severe inherited hemoglobinopathy with a large global burden, particularly in sub-Saharan Africa.1 Hydroxyurea, which is standard therapy for sickle cell anemia in high-resource settings, has had encouraging short-term results in low-resource settings.2,3 However, long-term safety and benefits remain undefined in Africa, where malnutrition, malaria, and poor health care access confound outcomes. Here, we summarize the use of hydroxyurea treatment in young Ugandan children with sickle cell anemia in a series of three studies over a 10-year period.
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