Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia—TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial

RCT in 121 children with abnormal transcranial doppler velocities (TCDVs) who have received ≥1 year of transfusions, and have no severe vasculopathy, found hydroxycarbamide was non-inferior to chronic transfusions for maintaining TCDVs and helping to prevent primary stroke.

At the first scheduled interim analysis, when non-inferiority was shown, the sponsor terminated the study. According to a commentary, these findings might help to fill some of the knowledge gaps that will enable care for children with sickle cell anaemia and elevated TCDVs in both low-income and high-income countries.