Hydroxyurea for the treatment of sickle cell disease: Efficacy, barriers, toxicity, and management in children

作者: John J. Strouse , Matthew M. Heeney

DOI: 10.1002/PBC.24178

关键词:

摘要: Hydroxyurea is the only approved medication in United States for treatment of sickle cell anemia (HbSS) and widely used children despite an indication limited to adults. We review evidence efficacy safety with reference pivotal adult studies. This expert opinion form basis recommended guidelines use hydroxyurea including indications, dosing, therapeutic monitoring, interventions improve adherence. However, there are substantial gaps our knowledge be addressed by on-going planned studies children.

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