TWiTCH: TCD With Transfusions Changing to Hydroxyurea for Sickle Cell Patients

Study title

TCD With Transfusions Changing to Hydroxyurea (TWiTCH): A Phase III randomized clinical trial to compare standard therapy (erythrocyte transfusions) with alternative therapy (hydroxyurea) for the maintenance of lowered TCD velocities in pediatric subjects with sickle cell anemia and abnormal pre-treatment TCD velocities

Study objectives

• Compare 24 months of alternative therapy (hydroxyurea) to standard therapy (transfusions) for the control of Transcranial Doppler Velocity (TCD V) in pediatric subjects with sickle cell anemia and abnormally high TCD V.
• Compare standard to alternative therapy regarding incidence of primary stroke, management of iron overload, effects on quality of life, frequency of non-stroke neurological events, frequency of other sickle cell-related events, and growth and development.

Eligibility

Patients with SCD (HbSS, HbSβ0 thalassemia, HbSOArab) who have had a previously documented index abnormal TCD examination and have had at least 12 months of chronic monthly erythrocyte transfusions since the index abnormal TCD. Ages 4.0 to 15.99 years at the time of enrollment.

PI: Dr. Janet Kwiatkowski

SC: Helen Stanley, Jolene Kokroko

Sponsor: National Institutes of Health