The primary objectives of this prospective study of hydroxyurea for children with sickle cell anemia are 1) Develop and prospectively evaluate a population pharmacokinetic/pharmacodynamics model to predict the maximum tolerated dose (MTD); 2) Identify urine biomarkers of hydroxyurea adherence using a novel metabolomics approach; 3) Identify pharmacogenomics modifiers of hydroxyurea MTD; and 4) Longitudinal monitoring of the effect of hydroxyurea upon organ function and quality of life.
| Amanda Pfeiffer, LPC, CCRP | |
| 513-803-4977 | |
| [email protected] |
| Adriane Hausfeld, RN,BSN,CCRP | |
| (513)803-3236 | |
| [email protected] |
6 Months - 21 Years
Not Applicable
Interventional
All
Drug
Diagnosis of sickle cell anemia (HbSS or Hbβ0-thalassemia)
Age 6 months to 21 years at the time of enrollment
Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy, including patients who are transitioning from chronic transfusions to hydroxyurea therapy
1. Family unwillingness to sign informed consent or comply with study treatments