Novel Dose Escalation to Predict Treatment With Hydroxyurea
Status:
Completed
Trial end date:
2020-07-31
Target enrollment:
Participant gender:
Summary
Sickle cell disease is a disorder in which red blood cells (RBCs) are abnormally shaped. This
can result in painful episodes, serious infections, chronic anemia (a decrease in the number
of red blood cells), and damage to body organs. Hydroxyurea therapy offers significant
benefits for infants, children, and adolescents with sickle cell anemia. These include a
reduction in the frequency of pain crises and acute chest syndrome (inflammation of the
lungs) and an increase in hemoglobin (the oxygen-carrying protein) in the blood. Patients on
hydroxyurea who receive a maximum tolerated dose (MTD) that is specific for them have greater
clinical benefit than those who receive a standard lower dose. There is, however, no way
currently to predict the MTD for individual patients. As such, MTD for each patient is
currently determined by gradual increases in the dose over several months. This process is
time-consuming, requires monthly clinic visits, and delays the benefits of hydroxyurea
therapy.
Our research group has come up with an equation that could be used to predict each patient's
MTD using baseline clinical and laboratory measures before starting hydroxyurea treatment.
The purpose of this research study is to compare the use of our equation for predicting MTD
to the current standard practice of gradually increasing the hydroxyurea dose until MTD is
reached. We want to see if the use of our predictive equation will allow us to achieve MTD
faster and with no more side effects than with the standard practice.
Phase:
Early Phase 1
Details
Lead Sponsor:
Baylor College of Medicine
Collaborators:
Children's Hospital Medical Center, Cincinnati Texas Children's Hospital