On October 23, 2013, the site team at St. Jude Children’s Research Hospital in Memphis, TN, enrolled the last patient into the PTN hydroxyurea study. Overall, 8 sites—Duke University Medical Center, Medical College of Wisconsin, Columbia University Medical Center, Ann & Robert H. Lurie Children’s Hospital of Chicago, Children’s of Alabama, University of Arkansas for Medical Sciences, University of Texas Southwestern, and St. Jude—worked very hard to enroll the 39 patients needed for this study.
Hydroxyurea represents the only major medical breakthrough in sickle cell disease in the past 20 years, and it is the only drug approved by the U.S. Food and Drug Administration (FDA) for use in adults with sickle cell. In spite of the fact that it is not labeled for use in children, hydroxyurea is often used to treat children who exhibit signs of severe sickle cell disease. Recent data suggest that its use in infants with sickle cell is feasible, well-tolerated, and efficacious; however, only limited pharmacokinetic and pharmacodynamic studies of hydroxyurea use in children exist.
This study was designed to better understand how this drug works in children and to confirm its safety profile. Participants were given capsules or a liquid formulation of hydroxyurea, and blood samples were obtained after administration to determine drug concentrations in the body and its elimination. Study results are expected by the first half of 2014.