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Hydroxyurea (HU) was introduced for the treatment of sickle cell disease (SCD) in 1984 and was approved by the U.S. FDA for this purpose in 1998. However, this drug is underused, for reasons that are unclear but perhaps related to lack of patient and physician awareness of its benefits, concerns about toxicities, or other factors. Therefore, the National Institutes of Health convened a consensus development conference in February 2008 to explore this topic. The participants have published an independent report that summarizes knowledge about HU as a treatment for SCD and suggests areas for future investigation.
The conference participants addressed five questions related to efficacy; use in various clinical situations; adverse effects; barri…