Children's Hospital Medical Center, Cincinnati
The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA. In this research study, the investigators hope to learn more about how to improve the dosing and monitoring of hydroxyurea and learn more about the long-term effects of hydroxyurea over time. Hydroxyurea is usually dosed based only on your weight. Our study will use a new way to select a starting dose that is based on how each patient absorbs hydroxyurea.
The EHANCE study will address key knowledge gaps about hydroxyurea for young children with SCA in nine innovative ways: * Novel functional assessments of brain, heart, kidneys, spleen, and eyes to assess organ protection in young children who receive hydroxyurea at MTD; * State-of-the-art assays to assess the benefits of hydroxyurea on growth, development, and reproductive health into puberty including serial measurements of pubertal development and sex hormones; * A simplified PK-guided strategy to optimize hydroxyurea initiation and dosing, with a long-term goal of validating pharmacogenomi…
Inclusion Criteria: * Diagnosis of sickle cell anemia (HbSS) or sickle-β0-thalassemia (HbSβ0) * Age 6 months at the time of enrollment * Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy Exclusion Criteria: * Current treatment with regularly scheduled blood transfusions * Sickle-hemoglobin C disease (HbSC), sickle-β+-thalassemia (HbSβ+)
Because people are different, we will measure how each participant's body absorbs and eliminates the medicine, hydroxyurea, using blood tests. This information will be used to determine the best dose for each participant (rather than using the same weight-based dose for everyone).
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