Stenosis Outcome At 1 And 3 Years After Transplantation Vs Standard-Care In Children With Sickle-Cell Anemia And Abnormal Transcranial Doppler With Stroke Or No-Stroke History

The presence of cerebral macrovasculopathy as detected by transcranial Doppler (TCD) exposes children with sickle cell anemia (SCA) to a high risk of stroke, preventable by chronic transfusion or stem cell transplantation (SCT). However, long-term outcomes of stenosis have not been well described. The Drepagreffe trial (NCT01340404) was a prospective trial comparing cerebral vasculopathy outcome after SCT vs standard-care in children with abnormal TCD with or without stroke history. Results from the whole population have recently been reported (Bernaudin et al, JAMA 2019). The decrease in velocities was significantly higher after SCT than standard-care (p<0.001) at 1 and 3-year, but the stenosis score was not different between both treatment groups. The aim of the present study was to determine stenosis outcome as a function of stroke or no-stroke history in both treatment groups using detailed post-hoc analysis.