The PTN hydroxyurea trial enrolled its 7th and 8th patients on June 12 and 14, 2012, thanks to the collective efforts of Nancy Green, MD, of Columbia University Medical Center, Robert Liem, MD, of Children’s Memorial Hospital in Chicago, and Courtney Thornburg, MD, of Duke University Medical Center.

Enrollment into this trial is proceeding in two stages: the first stage focused on children ages >5 – <17 years. The study protocol requires that, once the 8th patient is enrolled into this older cohort, the study team must submit an interim analysis of pharmacokinetic bioavailability and safety information to the Data and Safety Monitoring Board (DSMB). Once these data are reviewed, the DSMB will decide whether to allow enrollment of younger participants (ages 2 to ≤5 years) to begin. The DSMB plans to review these data on July 19, 2012.

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. Treatment with hydroxyurea has been shown to significantly decrease the incidence of painful episodes and also reduces overall mortality in adult patients. In spite of the fact that the FDA has not approved hydroxyurea for pediatric use, it is often used to treat children who exhibit signs of severe sickle cell disease. Recent data suggest that hydroxyurea therapy in infants with sickle cell is feasible, well-tolerated, and efficacious; however, only limited pharmacokinetic and pharmacodynamic studies of hydroxyurea use in children exist. The PTN is seeking to fill this knowledge gap.